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Records: 235
| Link to detailed view | Internal id - OD | SMPC ID (if available) | Orphan Designation status | Product Name | Active substance (Orphan Designation) | PL Number | Orphan Designation - indications | OD Expiry Date |
|---|---|---|---|---|---|---|---|---|
| See Detailed SMPC and OD Data | 145 | 301 | current | TAKHZYRO | lanadelumab Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein | PLGB 54937/0027 | TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older. | Nov. 26, 2030 |
| See Detailed SMPC and OD Data | 144 | 296 | current | TAKHZYRO | lanadelumab Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein | PLGB 54937/0020 | TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older. | Nov. 26, 2030 |
| See Detailed SMPC and OD Data | 143 | 293 | current | TAKHZYRO | lanadelumab Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein | PLGB 54937/0017 | TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older. | Nov. 26, 2030 |
| See Detailed SMPC and OD Data | 97 | 285 | current | Natpar | parathyroid hormone Recombinant human parathyroid hormone | PLGB 54937/0009 | Natpar is indicated as adjunctive treatment of adult patients with chronic hypoparathyroidism who cannot be adequately controlled with standard therapy alone. | April 26, 2027 |
| See Detailed SMPC and OD Data | 81 | 232 | current | LIVTENCITY | maribavir | PLGB 16189/0127 | Treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT). Consideration should be given to official guidance on the appropriate use of antiviral agents. | Nov. 11, 2032 |
| See Detailed SMPC and OD Data | 82 | 232 | current | LIVTENCITY | maribavir | PLGB 16189/0127 | Treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT). Consideration should be given to official guidance on the appropriate use of antiviral agents | Nov. 11, 2032 |
| See Detailed SMPC and OD Data | 67 | 220 | current | Ixazomib | ixazomib 2,2’-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid | PLGB 15475/0059 | Ixazomib in combination with lenalidomide and dexamethasone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy. | Nov. 23, 2026 |
| See Detailed SMPC and OD Data | 68 | current | Joenja | LENIOLISIB | PLGB 33010/0001 | Joenja is indicated for the treatment of activated phosphoinositide 3-kinase delta (P13K?) syndrome (APDS) in adult and paediatric patients 12 years of age and older. | Sept. 25, 2034 | |
| See Detailed SMPC and OD Data | 69 | current | Jorveza | budesonide | PLGB 08637/0030 | Jorveza is indicated for the treatment of eosinophilic esophagitis (EoE) in adults (older than 18 years of age). | Jan. 10, 2028 | |
| See Detailed SMPC and OD Data | 70 | current | Kaftrio | ivacaftor, tezacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide (elexacaftor) | PLGB 22352/0012 | Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (see section 5.1). | Aug. 21, 2030 | |
| See Detailed SMPC and OD Data | 71 | current | KIMMTRAK | tebentafusp | PLGB 36781/0001 | KIMMTRAK is indicated as monotherapy for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma. | June 6, 2032 | |
| See Detailed SMPC and OD Data | 72 | current | Kinpeygo | Budesonide | PLGB 17225/0027 | Treatment of primary immunoglobulin A (IgA) nephropathy (IgAN) in adults at risk of rapid disease progression with a urine protein-to-creatinine ratio (UPCR) ?1.5 g/gram | Feb. 1, 2033 | |
| See Detailed SMPC and OD Data | 73 | current | Koselugo | selumetinib hydrogen sulfate | PLGB 17901/0356 | Treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged 3 years and above. | Aug. 8, 2031 | |
| See Detailed SMPC and OD Data | 74 | current | Kymriah | tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | PLGB 00101/1101 | Kymriah is indicated for the treatment of paediatric and young adult patients up to 25 years of age with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post transplant or in second or later relapse. | Aug. 27, 2028 | |
| See Detailed SMPC and OD Data | 75 | current | Kymriah | tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | PLGB 00101/1101 | Kymriah is indicated for the treatment of: Adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy. | Aug. 27, 2028 | |
| See Detailed SMPC and OD Data | 76 | current | Kymriah | tisagenlecleucel Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 | PLGB 00101/1101 | Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy | Aug. 9, 2032 | |
| See Detailed SMPC and OD Data | 77 | current | Lamzede | velmanase alfa Recombinant human ?-Mannosidase | PLGB 08829/0188 | Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha mannosidosis. See sections 4.4 and 5.1. | March 27, 2028 | |
| See Detailed SMPC and OD Data | 78 | current | Ledaga | chlormethine | PLGB 12333/0016 | Ledaga is indicated for the topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF type CTCL) in adult patients | March 7, 2027 | |
| See Detailed SMPC and OD Data | 79 | current | Libmeldy | Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene | PLGB 49055/0002 | ibmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity: in children with late infantile or early juvenile forms, without clinical manifestations of the disease, in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline | Dec. 18, 2030 | |
| See Detailed SMPC and OD Data | 80 | current | LIVMARLI | maralixibat chloride | PLGB 56642/0001 | Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older | Feb. 10, 2035 | |
| See Detailed SMPC and OD Data | 83 | current | Loargys 5 mg/ml solution for injection/infusion | PEGZILARGINASE | PLGB 53487/0007 | Treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older. | Dec. 20, 2033 | |
| See Detailed SMPC and OD Data | 84 | current | Lonapegsomatropin Ascendis Pharma | lonapegsomatropin | PLGB 47497/0001 | Growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD]) | Oct. 17, 2032 | |
| See Detailed SMPC and OD Data | 85 | current | LUNSUMIO | mosunetuzumab | PLGB 00031/0933 | Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) who have received at least two prior systemic therapies | Oct. 4, 2032 | |
| See Detailed SMPC and OD Data | 86 | current | Lutathera | lutetium (177Lu) oxodotreotide Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl | PLGB 00101/1230 | Lutathera is indicated for the treatment of unresectable or metastatic, progressive, well differentiated (G1 and G2), somatostatin receptor positive gastroenteropancreatic neuroendocrine tumours (GEP NETs) in adults. | Sept. 28, 2027 | |
| See Detailed SMPC and OD Data | 87 | current | Luxturna | voretigene neparvovec | PLGB 00101/1104 | Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. | Dec. 5, 2028 | |
| See Detailed SMPC and OD Data | 88 | current | Luxturna | voretigene neparvovec | PLGB 00101/1104 | Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells. | Dec. 5, 2028 | |
| See Detailed SMPC and OD Data | 89 | current | Mepsevil | vestronidase alfa Recombinant human beta-glucuronidase | PLGB 47793/0002 | Mepsevil is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). | Aug. 27, 2028 | |
| See Detailed SMPC and OD Data | 90 | current | Minjuvi | tafasitamab | PLGB 42338/0016 | Minjuvi is indicated in combination with lenalidomide followed by Minjuvi monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) who are not eligible for autologous stem cell transplant (ASCT). | Oct. 7, 2031 | |
| See Detailed SMPC and OD Data | 91 | current | Myalepta | metreleptin | PLGB 08829/0215 | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed familial partial LD in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. | Aug. 1, 2028 | |
| See Detailed SMPC and OD Data | 92 | current | Myalepta | metreleptin | PLGB 08829/0215 | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. | Aug. 1, 2028 | |
| See Detailed SMPC and OD Data | 93 | current | Myalepta | metreleptin | PLGB 08829/0215 | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above | Aug. 1, 2028 | |
| See Detailed SMPC and OD Data | 94 | current | Myalepta | metreleptin | PLGB 08829/0215 | Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed congenital generalised LD (Berardinelli-Seip syndrome) | Aug. 1, 2028 | |
| See Detailed SMPC and OD Data | 95 | current | Mylotarg | gemtuzumab ozogamicin | PLGB 00057/1591 | MYLOTARG is indicated for combination therapy with daunorubicin (DNR) and cytarabine (AraC) for the treatment of patients age 15 years and above with previously untreated, de novo CD33 positive acute myeloid leukaemia (AML), except acute promyelocytic leukaemia (APL). | April 23, 2028 | |
| See Detailed SMPC and OD Data | 96 | current | Namuscla | mexiletine hydrochloride | PLGB 54289/0001 | Namuscla is indicated for the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders | Dec. 20, 2028 | |
| See Detailed SMPC and OD Data | 98 | current | Nexviadyme 100 mg powder for concentrate for solution for infusion | AVALGLUCOSIDASE ALFA | PLGB 04425/0893 | Long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid ?-glucosidase deficiency) | May 3, 2033 | |
| See Detailed SMPC and OD Data | 99 | current | Ngenla | somatrogon | PLGB 00057/1712 | Ngenla is indicated for the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone. | March 25, 2032 | |
| See Detailed SMPC and OD Data | 100 | current | Nulibry 9.5 mg powder for solution for injection | Fosdenopterin hydrobromide dihydrate | PLGB 24576/0009 | Nulibry is indicated for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A | April 9, 2036 | |
| See Detailed SMPC and OD Data | 101 | current | Ocaliva | obeticholic acid 6alpha-ethyl-chenodeoxycholic acid | PLGB 48025/0004 | Ocaliva is indicated for the treatment of primary biliary cholangitis (PCB) (also known as primary biliary cirrhosis) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA. | Dec. 15, 2026 | |
| See Detailed SMPC and OD Data | 102 | current | Ogsiveo 100mg film-coated tablets | Nirogacestat hydrobromide | PL 59359/0001 | Ogsiveo as monotherapy is indicated for the treatment of adult patients with progressing desmoid tumours who require systemic treatment | Jan. 7, 2036 | |
| See Detailed SMPC and OD Data | 103 | current | Ogsiveo 150mg film-coated tablets | Nirogacestat hydrobromide | PL 59359/0002 | Ogsiveo as monotherapy is indicated for the treatment of adult patients with progressing desmoid tumours who require systemic treatment | Jan. 7, 2036 | |
| See Detailed SMPC and OD Data | 104 | current | Onivyde pegylated liposomal | irinotecan Nanoliposomal irinotecan | PLGB 05815/0111 | In combination with oxaliplatin, 5?fluorouracil (5?FU) and leucovorin (LV) for the first-line treatment of adult patients with metastatic adenocarcinoma of the pancreas, in combination with 5-FU and LV for the treatment of metastatic adenocarcinoma of the pancreas, in combination with 5?fluorouracil (5?FU) and leucovorin (LV), in adult patients who have progressed following gemcitabine based therapy. | Oct. 18, 2026 | |
| See Detailed SMPC and OD Data | 105 | current | Onpattro | patisiran Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA | PLGB 50597/0002 | Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. | Aug. 29, 2028 | |
| See Detailed SMPC and OD Data | 106 | current | Opfolda | MIGLUSTAT | PLGB 25823/0004 | Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ??glucosidase [GAA] deficiency). | Aug. 8, 2033 | |
| See Detailed SMPC and OD Data | 107 | current | OXBRYTA | voxelotor | PLGB 00057/1720 | Oxbryta is indicated for the treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide. | July 25, 2032 | |
| See Detailed SMPC and OD Data | 108 | current | Oxlumo | lumasiran Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues | PLGB 50597/0005 | Oxlumo is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in all age groups. | Nov. 23, 2030 | |
| See Detailed SMPC and OD Data | 109 | current | Pemazyre | pemigatinib | PLGB 42338/0008 | The treatment of adults with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy. | April 7, 2031 | |
| See Detailed SMPC and OD Data | 110 | current | Polivy | polatuzumab vedotin | PLGB 00031/0912 | Polivy in combination with rituximab, cyclophosphamide, doxorubicin, and prednisone (R-CHP) is indicated for the treatment of adult patients with previously untreated diffuse large B-cell lymphoma (DLBCL). Polivy in combination with bendamustine and rituximab is indicated for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant. | Jan. 20, 2030 | |
| See Detailed SMPC and OD Data | 111 | current | Pombiliti 105 mg powder for concentrate for solution for infusion | CIPAGLUCOSIDASE ALFA | PLGB 25823/0003 | Authorised orphan indication: Long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid ?-glucosidase [GAA] deficiency | June 30, 2033 | |
| See Detailed SMPC and OD Data | 112 | current | Poteligeo | mogamulizumab | PLGB 16508/0074 | POTELIGEO is indicated for the treatment of adult patients with mycosis fungoides (MF) or Sézary syndrome (SS) who have received at least one prior systemic therapy. | Nov. 26, 2028 | |
| See Detailed SMPC and OD Data | 113 | current | PREVYMIS 240 mg concentrate for solution for infusion | Letermovir | PLGB 53095/0046 | PREVYMIS is indicated for prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult and paediatric patients weighing at least 5 kg who are CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT). PREVYMIS is indicated for prophylaxis of CMV disease in CMV-seronegative adults and paediatric patients weighing at least 40 kg who have received a kidney transplant from a CMV-seropositive donor [D+/R-]. Consideration should be given to official guidance on the appropriate use of antiviral agents. | Jan. 10, 2030 | |
| See Detailed SMPC and OD Data | 114 | current | PREVYMIS 240 mg film-coated tablets | Letermovir | PLGB 53095/0048 | PREVYMIS is indicated for prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult and paediatric patients weighing at least 15 kg who are CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT). PREVYMIS is indicated for prophylaxis of CMV disease in CMV-seronegative adults and paediatric patients weighing at least 40 kg who have received a kidney transplant from a CMV-seropositive donor [D+/R-]. Consideration should be given to official guidance on the appropriate use of antiviral agents. | Jan. 10, 2030 | |
| See Detailed SMPC and OD Data | 115 | current | PREVYMIS 20 mg granules in sachet | Letermovir | PL 53095/0110 | PREVYMIS is indicated for prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult and paediatric patients weighing at least 5 kg who are CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT). PREVYMIS is indicated for prophylaxis of CMV disease in CMV-seronegative adults and paediatric patients weighing at least 40 kg who have received a kidney transplant from a CMV-seropositive donor [D+/R-]. Consideration should be given to official guidance on the appropriate use of antiviral agents. | Jan. 10, 2030 | |
| See Detailed SMPC and OD Data | 116 | current | PREVYMIS 120 mg granules in sachet | Letermovir | PL 53095/0111 | PREVYMIS is indicated for prophylaxis of cytomegalovirus (CMV) reactivation and disease in adult and paediatric patients weighing at least 5 kg who are CMV-seropositive recipients [R+] of an allogeneic haematopoietic stem cell transplant (HSCT). PREVYMIS is indicated for prophylaxis of CMV disease in CMV-seronegative adults and paediatric patients weighing at least 40 kg who have received a kidney transplant from a CMV-seropositive donor [D+/R-]. Consideration should be given to official guidance on the appropriate use of antiviral agents. | Jan. 10, 2030 | |
| See Detailed SMPC and OD Data | 117 | current | PYRUKYND | Mitapivat sulfate Potent, allosteric activator of wild-type red blood cell (RBC)-specific form of pyruvate kinase (PKR) and a range of mutant PKR enzymes. Mitapivat targets the underlying enzymatic defect that causes haemolysis in pyruvate kinase deficiency by restoring the activity of mutant forms of PKR. | PLGB 52779/0001 | Pyrukynd is indicated for the treatment of pyruvate kinase deficiency (PK deficiency) in adult patients | Nov. 29, 2032 | |
| See Detailed SMPC and OD Data | 118 | current | Qalsody | Tofersen | PL 22407/0034 | Treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 (SOD1) gene | July 22, 2035 | |
| See Detailed SMPC and OD Data | 119 | current | Qarziba | dinutuximab beta Chimeric monoclonal antibody against GD2 | PLGB 44185/0005 | Qarziba is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilised by other suitable measures. In patients with a history of relapsed/refractory disease and in patients who have not achieved a complete response after first line therapy, Qarziba should be combined with interleukin-2 (IL-2). | May 11, 2027 | |
| See Detailed SMPC and OD Data | 120 | current | QINLOCK | ripretinib | PLGB 55295/0004 | QINLOCK is indicated for the treatment of adult patients with advanced gastrointestinal stromal tumour (GIST) who have received prior treatment with three or more kinase inhibitors, including imatinib | Dec. 20, 2031 | |
| See Detailed SMPC and OD Data | 121 | current | Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | PLGB 53487/0001 | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | Dec. 1, 2027 | |
| See Detailed SMPC and OD Data | 122 | current | Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | PLGB 53487/0001 | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | Dec. 1, 2027 | |
| See Detailed SMPC and OD Data | 123 | current | Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | PLGB 53487/0001 | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | Dec. 1, 2027 | |
| See Detailed SMPC and OD Data | 124 | current | Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | PLGB 53487/0001 | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | Dec. 1, 2027 | |
| See Detailed SMPC and OD Data | 125 | current | Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | PLGB 53487/0001 | RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | Dec. 1, 2027 | |
| See Detailed SMPC and OD Data | 126 | current | Ravicti | Glycerol phenylbutyrate Glyceryl tri-(4-phenylbutyrate) | PLGB 53487/0001 | Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome): RAVICTI is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements). | Dec. 1, 2027 | |
| See Detailed SMPC and OD Data | 127 | current | Reblozyl | luspatercept Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain | PLGB 15105/0153 | In adults for the treatment of transfusion-dependent anaemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS). | June 26, 2030 | |
| See Detailed SMPC and OD Data | 128 | current | REZUROCK | belumosudil mesilate | PLGB 04425/0902 | Rezurock is indicated for the treatment of patients aged 12 years and older with chronic graft-versus-host disease (chronic GVHD) who have received at least two prior lines of systemic therapy. | July 6, 2032 | |
| See Detailed SMPC and OD Data | 129 | current | Rezzayo | REZAFUNGIN ACETATE | PLGB 16950/0390 | Treatment of invasive candidiasis in adult | Jan. 29, 2034 | |
| See Detailed SMPC and OD Data | 130 | current | Rydapt | midostaurin | PLGB 00101/1130 | Rydapt is indicated in combination with standard daunorubicin and cytarabine induction and high dose cytarabine consolidation chemotherapy, and for patients in complete response followed by Rydapt single agent maintenance therapy, for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are FLT3 mutation positive | Sept. 20, 2027 | |
| See Detailed SMPC and OD Data | 131 | current | Rydapt | midostaurin | PLGB 00101/1130 | Rydapt is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated haematological neoplasm (SM AHN), or mast cell leukaemia (MCL). | Sept. 20, 2027 | |
| See Detailed SMPC and OD Data | 132 | current | Rystiggo 140 mg/ml solution for injection | ROZANOLIXIZUMAB | PLGB 00039/0808 | as an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. | March 7, 2034 | |
| See Detailed SMPC and OD Data | 133 | current | Scemblix | Asciminib | PLGB 00101/1207 | Scemblix is indicated for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase, previously treated with two or more tyrosine kinase inhibitors, and without a known T315I mutation | June 15, 2032 | |
| See Detailed SMPC and OD Data | 134 | current | Skyclarys 50 mg hard capsules | OMAVELOXOLONE | PLGB 22407/0033 | treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. | April 23, 2035 | |
| See Detailed SMPC and OD Data | 135 | current | Sogroya | somapacitan | PLGB 04668/0434 | Sogroya is indicated for the replacement of endogenous growth hormone (GH) in children aged 3 years and above, and adolescents with growth failure due to growth hormone deficiency (paediatric GHD), and in adults with growth hormone deficiency (adult GHD). | Oct. 21, 2031 | |
| See Detailed SMPC and OD Data | 136 | current | Soliris | eculizumab | PLGB 31187/0020 | Soliris is indicated for the treatment of refractory generalized myasthenia gravis (gMG) in patients aged 6 years and above who are antiacetylcholine receptor (AChR) antibody-positive | Aug. 17, 2027 | |
| See Detailed SMPC and OD Data | 137 | current | Soliris | eculizumab | PLGB 31187/0020 | Soliris is indicated in adults for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in patients who are anti-aquaporin-4 (AQP4) antibody-positive with a relapsing course of the disease. | Aug. 28, 2029 | |
| See Detailed SMPC and OD Data | 138 | current | SomaKit TOC | edotreotide Gallium (68Ga)-edotreotide | PLGB 00101/1229 | After radiolabelling with gallium (68Ga) chloride solution, the solution of gallium (68Ga) edotreotide obtained is indicated for Positron Emission Tomography (PET) imaging of somatostatin receptor overexpression in adult patients with confirmed or suspected well-differentiated gastro-enteropancreatic neuroendocrine tumours (GEP-NET) for localizing primary tumours and their metastases. | Dec. 12, 2026 | |
| See Detailed SMPC and OD Data | 139 | current | Spexotras | Trametinib | PLGB 00101/1227 | Low-grade Glioma: Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. High-grade glioma Spexotras in combination with dabrafenib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment. | Feb. 13, 2036 | |
| See Detailed SMPC and OD Data | 140 | current | Spinraza | nusinersen Antisense oligonucleotide targeted to the SMN2 gene | PLGB 22407/0018 | Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy. | June 1, 2029 | |
| See Detailed SMPC and OD Data | 141 | current | Strimvelis | autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence | PLGB 58042/0001 | Strimvelis is indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. | May 30, 2028 | |
| See Detailed SMPC and OD Data | 142 | current | Symkevi | tezacaftor/ivacaftor | PLGB 22352/0003 | Symkevi is indicated in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A?G, S945L, S977F, R1070W, D1152H, 2789+5G?A, 3272 26A?G, and 3849+10kbC?T. | Nov. 6, 2028 | |
| See Detailed SMPC and OD Data | 146 | current | TAKHZYRO | lanadelumab Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein | PLGB 54937/0028 | TAKHZYRO is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 2 years and older. | Nov. 26, 2030 | |
| See Detailed SMPC and OD Data | 147 | current | Tecartus | autologous anti-CD19-transduced CD3+ cells | PLGB 11972/0045 | Tecartus is indicated for the treatment of adult patients 26 years of age and above with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL). | Nov. 7, 2032 | |
| See Detailed SMPC and OD Data | 148 | current | Tecartus | autologous anti-CD19-transduced CD3+ cells | PLGB 11972/0045 | Tecartus is indicated for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor. | Dec. 14, 2030 | |
| See Detailed SMPC and OD Data | 149 | current | Tegsedi | inotersen | PLGB 51704/0002 | Tegsedi is indicated for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). | July 10, 2028 | |
| See Detailed SMPC and OD Data | 150 | current | TIBSOVO | ivosidenib | PLGB 05815/0120 | In combination with azacitidine for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) with an isocitrate dehydrogenase-1 (IDH1) R132 mutation who are not eligible to receive standard induction chemotherapy (see section 5.1) | July 5, 2033 | |
| See Detailed SMPC and OD Data | 151 | current | TIBSOVO | ivosidenib | PLGB 05815/0120 | as monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy (see section 5.1). | July 5, 2033 | |
| See Detailed SMPC and OD Data | 152 | current | Trecondi | treosulfan | PLGB 11587/0118 | Treosulfan in combination with fludarabine is indicated as part of conditioning treatment prior to allogeneic haematopoietic stem cell transplantation (alloHSCT) in adult patients and in paediatric patients older than one month with malignant and non malignant diseases. | June 24, 2031 | |
| See Detailed SMPC and OD Data | 153 | current | Trepulmix | treprostinil sodium | PLGB 51174/0001 | Trepulmix is indicated for the treatment of adult patients with WHO Functional Class (FC) III or IV and: inoperable chronic thromboembolic pulmonary hypertension (CTEPH), or persistent or recurrent CTEPH after surgical treatment to improve exercise capacity. | April 7, 2030 | |
| See Detailed SMPC and OD Data | 154 | current | UPSTAZA | eladocagene exuparvovec | PLGB 44221/0006 | For the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype. | Nov. 17, 2034 | |
| See Detailed SMPC and OD Data | 155 | current | Vanflyta | Quizartinib | PLGB 08265/0047 | In combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by VANFLYTA single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3-ITD positive | March 11, 2034 | |
| See Detailed SMPC and OD Data | 156 | current | Verkazia | ciclosporin | PLGB 16058/0028 | Treatment of severe vernal keratoconjunctivitis (VKC) in children from 4 years of age and adolescents | July 10, 2030 | |
| See Detailed SMPC and OD Data | 157 | current | Voranigo 10 mg film-coated tablets | VORASIDENIB CITRATE | PL 05815/0122 | Treatment of Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation or isocitrate dehydrogenase-2 (IDH2) mutation in adults and paediatric patients 12 years and older, who are not in need of immediate chemotherapy or radiotherapy following surgical intervention | Sept. 16, 2037 | |
| See Detailed SMPC and OD Data | 158 | current | Voranigo 40 mg film-coated tablets | VORASIDENIB CITRATE | PL 05815/0123 | Treatment of Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation or isocitrate dehydrogenase-2 (IDH2) mutation in adults and paediatric patients 12 years and older, who are not in need of immediate chemotherapy or radiotherapy following surgical intervention | Sept. 16, 2037 | |
| See Detailed SMPC and OD Data | 159 | current | Voraxaze 1000 units powder for solution for injection | GLUCARPIDASE | PLGB 18442/0002 | Voraxaze is indicated to reduce toxic plasma methotrexate concentration in adults and children (aged 28 days and older) with delayed methotrexate elimination. | June 15, 2035 | |
| See Detailed SMPC and OD Data | 160 | current | Vyloy | zolbetuximab | PLGB 00166/0439 | Vyloy, in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 -negative gastric or gastro-oesophageal junction adenocarcinoma whose tumours are Claudin 18.2 positive | Aug. 14, 2034 | |
| See Detailed SMPC and OD Data | 161 | current | Vyndaqel | tafamidis N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate | PLGB 00057/1660 | Treatment of wild-type transthyretin amyloidosis in adult patients with cardiomyopathy | Feb. 19, 2030 | |
| See Detailed SMPC and OD Data | 162 | current | Vyvgart 20 mg/ml Concentrate for solution for infusion | efgartigimod alfa | PLGB 47104/0004 | Treatment of adult patients with generalised Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. | March 14, 2033 | |
| See Detailed SMPC and OD Data | 163 | current | Vyvgart 1000 mg solution for injection) | efgartigimod alfa | PLGB 47104/0005 | Treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins | Nov. 25, 2035 | |
| See Detailed SMPC and OD Data | 164 | current | Vyvgart 1000 mg solution for injection in pre-filled syringe | efgartigimod alfa | PL 47104/0006 | Treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins | Nov. 25, 2035 | |
| See Detailed SMPC and OD Data | 165 | current | Vyxeos liposomal | daunorubicin / cytarabine Liposomal combination of cytarabine and daunorubicin | PLGB 31626/0004 | Vyxeos is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). | Aug. 27, 2028 | |
| See Detailed SMPC and OD Data | 166 | current | Wainzua | EPLONTERSEN | PLGB 17901/0377 | Authorised orphan indication: Wainzua is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv amyloidosis) in adult patients with Stage 1 and 2 polyneuropathy. | Oct. 14, 2034 | |
| See Detailed SMPC and OD Data | 167 | current | Waylivra | volanesorsen Phosphorothioate oligonucleotide targeted to apolipoprotein C-III | PLGB 51704/0003 | Waylivra is indicated as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate | May 8, 2029 | |
| See Detailed SMPC and OD Data | 168 | current | Winrevair | Sotatercept | PLGB 53095/0104 | Winrevair, in combination with other pulmonary arterial hypertension (PAH) therapies, is indicated for the treatment of PAH in adult patients with WHO Functional Class (FC) II to III, to improve exercise capacity. | Dec. 27, 2034 | |
| See Detailed SMPC and OD Data | 169 | current | XENPOZYME | Olipudase alfa | PLGB 04425/0901 | Enzyme replacement therapy for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency in paediatric and adult patients with type A/B or type B. | July 31, 2032 | |
| See Detailed SMPC and OD Data | 170 | current | Xermelo | telotristat (S)-ethyl 2-amino-3-(4-(2-amino-6-((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate | PLGB 26080/0009 | Xermelo is indicated for the treatment of carcinoid syndrome diarrhoea in combination with somatostatin analogue (SSA) therapy in adults inadequately controlled by SSA therapy. | Sept. 20, 2027 | |
| See Detailed SMPC and OD Data | 171 | current | Yescarta | axicabtagene ciloleucel | PLGB 11972/0044 | Treatment of adult patients with diffuse large B cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) that relapses within 12 months from completion of, or is refractory to, first-line chemoimmunotherapy. Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy | Aug. 27, 2028 | |
| See Detailed SMPC and OD Data | 172 | current | Yescarta | axicabtagene ciloleucel | PLGB 11972/0044 | YESCARTA is indicated for the treatment of adult patients with relapsed or refractory primary mediastinal large B-cell lymphoma (PMBCL) after two or more lines of systemic therapy. | Aug. 27, 2028 | |
| See Detailed SMPC and OD Data | 173 | current | Yescarta | axicabtagene ciloleucel | PLGB 11972/0044 | Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after three or more lines of systemic therapy’ | Aug. 9, 2032 | |
| See Detailed SMPC and OD Data | 174 | current | Yorvipath 420 micrograms/1.4 mL solution for injection in pre filled pen | PALOPEGTERIPARATIDE | PLGB 51127/0001 | Treatment of hypoparathyroidism. | April 23, 2034 | |
| See Detailed SMPC and OD Data | 175 | current | Zejula | niraparib (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt | PLGB 19494/0294 | as monotherapy for the maintenance treatment of adult patients with advanced epithelial (FIGO Stages III and IV) high-grade ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy. as monotherapy for the maintenance treatment of adult patients with platinum sensitive relapsed high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum based chemotherapy | Nov. 20, 2027 | |
| See Detailed SMPC and OD Data | 176 | current | Zokinvy | lonafarnib | PLGB 24576/0011 | Treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation. | Aug. 24, 2034 | |
| See Detailed SMPC and OD Data | 177 | current | Zolgensma | onasemnogene abeparvovec Adeno-associated viral vector serotype 9 containing the human SMN gene | PLGB 00101/1223 | patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene. | May 18, 2030 | |
| See Detailed SMPC and OD Data | 178 | current | Ztalmy 50 mg/ml Oral suspension | GANAXOLONE | PL 53487/0010 | indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older. | March 7, 2034 | |
| See Detailed SMPC and OD Data | 179 | expired | ADAKVEO | crizanlizumab | PLGB 00101/1191 | Adakveo is indicated for the prevention of recurrent vaso-occlusive crises (VOCs) in sickle cell disease patients aged 16 years and older. It can be given as an add on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is inappropriate or inadequate. | Oct. 29, 2030 | |
| See Detailed SMPC and OD Data | 180 | expired | ADCETRIS | brentuximab vedotin, Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E | PLGB 16189/0093 | ADCETRIS in combination with cyclophosphamide, doxorubicin and prednisone (CHP) is indicated for adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL). ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory sALCL. | Oct. 30, 2024 | |
| See Detailed SMPC and OD Data | 181 | expired | ADCETRIS | brentuximab vedotin, Monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E | PLGB 16189/0093 | ADCETRIS is indicated for adult patients with previously untreated CD30+ Stage IV Hodgkin lymphoma (HL) in combination with doxorubicin, vinblastine and dacarbazine (AVD). ADCETRIS is indicated for the treatment of adult patients with CD30+ HL at increased risk of relapse or progression following autologous stem cell transplant (ASCT). ADCETRIS is indicated for the treatment of adult patients with relapsed or refractory CD30+ Hodgkin lymphoma (HL): 1. following ASCT, or 2. following at least two prior therapies when ASCT or multi-agent chemotherapy is not a treatment option. | Oct. 30, 2024 | |
| See Detailed SMPC and OD Data | 182 | expired | Adempas | riociguat, Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate | PLGB 00010/0670 | Adempas is indicated for the treatment of adult patients with WHO Functional Class (FC) II to III with - inoperable CTEPH - persistent or recurrent CTEPH after surgical treatment, to improve exercise capacity. Pulmonary arterial hypertension (PAH) Adempas, as monotherapy or in combination with endothelin receptor antagonists, is indicated for the treatment of adult patients with pulmonary arterial hypertension (PAH) with WHO Functional Class (FC) II to III to improve exercise capacity. Efficacy has been shown in a PAH population including aetiologies of idiopathic or heritable PAH or PAH associated with connective tissue disease. | March 31, 2024 | |
| See Detailed SMPC and OD Data | 183 | expired | AMVUTTRA | Vutrisiran Sodium | PLGB 50597/0006 | Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. | Sept. 16, 2032 | |
| See Detailed SMPC and OD Data | 184 | expired | Bronchitol | Mannitolum | PLGB 27944/0002 | Bronchitol is indicated for the treatment of cystic fibrosis (CF) in adults aged 18 years and above as an add-on therapy to best standard of care. | April 18, 2022 | |
| See Detailed SMPC and OD Data | 185 | expired | Carbaglu | Carglumic acid | PLGB 15266/0019 | Carbaglu is indicated in treatment of hyperammonaemia due to isovaleric acidaemia. | June 1, 2021 | |
| See Detailed SMPC and OD Data | 186 | expired | Carbaglu | Carglumic acid | PLGB 15266/0019 | Carbaglu is indicated in treatment of hyperammonaemia due to methymalonic acidaemia. | June 1, 2021 | |
| See Detailed SMPC and OD Data | 187 | expired | Carbaglu | Carglumic acid | PLGB 15266/0019 | Carbaglu is indicated in treatment of hyperammonaemia due to propionic acidaemia. | June 1, 2021 | |
| See Detailed SMPC and OD Data | 188 | expired | Cerdelga | eliglustat | PLGB 04425/0763 | Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs). | Jan. 21, 2025 | |
| See Detailed SMPC and OD Data | 189 | expired | Columvi 2.5 mg and 10mg concentrate for solution for infusion | GLOFITAMAB | PLGB 00031/0931 | Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL), after two or more lines of systemic therapy. | Oct. 16, 2033 | |
| See Detailed SMPC and OD Data | 190 | expired | Cometriq | cabozantinib, Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt | PLGB 28247/0004 | COMETRIQ is indicated for the treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma. For patients in whom rearranged during transfection (RET) mutation status is not known or is negative, a possible lower benefit should be taken into account before individual treatment decision. | March 26, 2024 | |
| See Detailed SMPC and OD Data | 191 | expired | Dacogen | decitabine | PLGB 00242/0662 | Dacogen is indicated for the treatment of adult patients with newly diagnosed de novo or secondary acute myeloid leukaemia (AML), according to the World Health Organisation (WHO) classification, who are not candidates for standard induction chemotherapy. | Sept. 24, 2024 | |
| See Detailed SMPC and OD Data | 192 | expired | Defitelio | defibrotide | PLGB 31626/0005 | Treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy. It is indicated in adults and in adolescents, children and infants over 1 month of age. | Oct. 22, 2023 | |
| See Detailed SMPC and OD Data | 193 | expired | Esbriet | pirfenidone | PLGB 00031/0851 | Esbriet is indicated in adults for the treatment of mild to moderate Idiopathic Pulmonary Fibrosis (IPF). | March 2, 2021 | |
| See Detailed SMPC and OD Data | 194 | expired | Evrysdi | risdiplam | PLGB 00031/0920 | Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. | May 19, 2031 | |
| See Detailed SMPC and OD Data | 195 | expired | Farydak | panobinostat | PLGB 54599/0008 | Farydak, in combination with bortezomib and dexamethasone, is indicated for the treatment of adult patients with relapsed and/or refractory multiple myeloma who have received at least two prior regimens including bortezomib and an immunomodulatory agent. | Sept. 1, 2025 | |
| See Detailed SMPC and OD Data | 196 | expired | Gazyvaro | obinutuzumab | PLGB 00031/0856 | Gazyvaro in combination with chlorambucil is indicated for the treatment of adult patients with previously untreated CLL and with comorbidities making them unsuitable for full-dose fludarabine based therapy (see section 5.1). | July 24, 2024 | |
| See Detailed SMPC and OD Data | 197 | expired | Gazyvaro | obinutuzumab | PLGB 00031/0856 | Gazyvaro in combination with chemotherapy, followed by Gazyvaro maintenance therapy in patients achieving a response, is indicated for the treatment of patients with previously untreated advanced FL (see section 5.1) Gazyvaro in combination with bendamustine followed by Gazyvaro maintenance is indicated for the treatment of patients with FL who did not respond or who progressed during or up to 6 months after treatment with rituximab or a rituximab-containing regimen. | June 15, 2026 | |
| See Detailed SMPC and OD Data | 198 | expired | Granupas | Para-aminosalicylic acid | PLGB 35068/0003 | Indicated for use as part of an appropriate combination regimen for multi-drug resistant tuberculosis in adults and paediatric patients from 28 days of age and older when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability. Consideration should be given to official guidance on the appropriate use of antibacterial agents. | April 9, 2024 | |
| See Detailed SMPC and OD Data | 199 | expired | Hepcludex | bulevirtide | PLGB 50662/0002 | The treatment of chronic hepatitis delta virus (HDV) infection in plasma (or serum) HDV-RNA positive adult patients with compensated liver disease. | Aug. 5, 2030 | |
| See Detailed SMPC and OD Data | 200 | expired | Hetlioz | tasimelteon | PLGB 54231/0001 | HETLIOZ is indicated for the treatment of Non-24-Hour Sleep-Wake Disorder (Non-24) in totally blind adults. | July 7, 2025 | |
| See Detailed SMPC and OD Data | 201 | expired | Iclusig | ponatinib Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] | PLGB 49635/0001 | Iclusig is indicated in adult patients with Philadelphia chromosome positive acute lymphoblastic leukaemia (Ph+ ALL) who are resistant to dasatinib; who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation. | July 3, 2023 | |
| See Detailed SMPC and OD Data | 202 | expired | Iclusig | ponatinib Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] | PLGB 49635/0001 | Iclusig is indicated in adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukaemia (CML) who are resistant to dasatinib or nilotinib; who are intolerant to dasatinib or nilotinib and for whom subsequent treatment with imatinib is not clinically appropriate; or who have the T315I mutation | July 3, 2023 | |
| See Detailed SMPC and OD Data | 203 | expired | Imnovid | pomalidomide | PLGB 50412/0002 | Imnovid in combination with dexamethasone is indicated in the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior treatment regimens, including both lenalidomide and bortezomib, and have demonstrated disease progression on the last therapy | Aug. 8, 2023 | |
| See Detailed SMPC and OD Data | 204 | expired | Kalydeco | ivacaftor N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide | PLGB 22352/0008 | Treatment of cystic fibrosis (CF) in patients age six years and older who have a G551D mutation in the CFTR gene | July 25, 2022 | |
| See Detailed SMPC and OD Data | 205 | expired | Kalydeco | ivacaftor N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide | PLGB 22352/0008 | In a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1) | July 25, 2022 | |
| See Detailed SMPC and OD Data | 206 | expired | Kalydeco | ivacaftor N-(2,4-Di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide | PLGB 22352/0013 | In a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1) | July 25, 2022 | |
| See Detailed SMPC and OD Data | 207 | expired | Kanuma | sebelipase alfa Recombinant human lysosomal acid lipase | PLGB 31187/0017 | Kanuma is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) Deficiency. | Sept. 1, 2025 | |
| See Detailed SMPC and OD Data | 208 | expired | Ketoconazole HRA | Ketoconazole | PLGB 51757/0002 | Ketoconazole HRA is indicated for the treatment of endogenous Cushing’s syndrome in adults and adolescents above the age of 12 years. | Nov. 21, 2024 | |
| See Detailed SMPC and OD Data | 209 | expired | Kyprolis | carfilzomib | PLGB 13832/0023 | Kyprolis in combination with daratumumab and dexamethasone, with lenalidomide and dexamethasone, or with dexamethasone alone is indicated for the treatment of adult patients with multiple myeloma who have received at least one prior therapy. | Nov. 23, 2025 | |
| See Detailed SMPC and OD Data | 210 | expired | Mozobil | plerixafor l, 1’-[1,4-phenylenebis (methylene)]-bis-1,4,8,11- tetraazacyclotetradecane | PLGB 04425/0769 | Adult patients Mozobil is indicated in combination with granulocyte-colony stimulating factor (G-CSF) to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in adult patients with lymphoma or multiple myeloma whose cells mobilise poorly. Paediatric patients (1 to less than 18 years) Mozobil is indicated in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children with lymphoma or solid malignant tumours, either: - pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilization with G-CSF (with or without chemotherapy) is expected to be insufficient with regards to desired hematopoietic stem cells yield, or - who previously failed to collect sufficient haematopoietic stem cells. | Aug. 4, 2021 | |
| See Detailed SMPC and OD Data | 211 | expired | Nexavar | sorafenib tosylate | PLGB 00010/0701 | Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (follicular/Hürthle cell) thyroid carcinoma, refractory to radioactive iodine. | May 27, 2024 | |
| See Detailed SMPC and OD Data | 212 | expired | Nexavar | sorafenib tosylate | PLGB 00010/0701 | Nexavar is indicated for the treatment of patients with progressive, locally advanced or metastatic, differentiated (papillary) thyroid carcinoma, refractory to radioactive iodine. | May 27, 2024 | |
| See Detailed SMPC and OD Data | 213 | expired | NexoBrid | Concentrate of proteolytic enzymes enriched in bromelain Purified bromelain | PLGB 43948/0002 | NexoBrid is indicated for removal of eschar in adults with deep partial- and full-thickness thermal burns. | Dec. 20, 2022 | |
| See Detailed SMPC and OD Data | 214 | expired | Obiltoxaximab SFL | Obiltoxaximab is a monoclonal antibody that binds the protective antigen (PA) of B. anthracis | PLGB 54280/0001 | Obiltoxaximab SFL is indicated in combination with appropriate antibacterial drugs in all age groups for treatment of inhalational anthrax due to Bacillus anthracis (see section 5.1). Obiltoxaximab SFL is indicated in all age groups for post-exposure prophylaxis of inhalational anthrax when alternative therapies are not appropriate or are not available (see section 5.1). | Nov. 19, 2030 | |
| See Detailed SMPC and OD Data | 215 | expired | Opsumit | macitentan | PLGB 00242/0663 | Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III. Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease. | Dec. 27, 2023 | |
| See Detailed SMPC and OD Data | 216 | expired | Plenadren | hydrocortisone | PLGB 39534/0009 | Treatment of adrenal insufficiency in adults. | Nov. 14, 2021 | |
| See Detailed SMPC and OD Data | 217 | expired | Raxone | idebenone | PLGB 08829/0204 | Raxone is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). | Sept. 10, 2025 | |
| See Detailed SMPC and OD Data | 218 | expired | Revestive | teduglutide [gly2] Recombinant human glucagon-like peptide | PLGB 16189/0077 | Revestive is indicated for the treatment of patients aged 1 year and above with Short Bowel Syndrome. Patients should be stable following a period of intestinal adaptation after surgery. | Sept. 4, 2024 | |
| See Detailed SMPC and OD Data | 219 | expired | SCENESSE | afamelanotide [Nle4, D-Phe7]-alpha-melanocyte stimulating hormone | PLGB 30619/0002 | Scenesse is indicated for prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP). | Dec. 29, 2024 | |
| See Detailed SMPC and OD Data | 220 | expired | Signifor | pasireotide | PLGB 15266/0032 | Treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative and who are inadequately controlled on treatment with another somatostatin analogue. The 60 mg strength is only to be used in the treatment of acromegaly. | Nov. 21, 2024 | |
| See Detailed SMPC and OD Data | 221 | expired | Signifor | pasireotide | PLGB 15266/0032 | Treatment of adult patients with Cushing’s disease for whom surgery is not an option or for whom surgery has failed. | April 27, 2022 | |
| See Detailed SMPC and OD Data | 222 | expired | Sirturo 20 mg Tablets | BEDAQUILINE FUMARATE | PLGB 00242/0744 | SIRTURO is indicated for use as part of an appropriate combination regimen in adult and paediatric patients (5 years to less than 18 years of age and weighing at least 15 kg) with pulmonary tuberculosis (TB) due to Mycobacterium tuberculosis resistant to at least rifampicin and isoniazid. | March 7, 2024 | |
| See Detailed SMPC and OD Data | 223 | expired | Soliris | eculizumab | PLGB 31775/0003 | Soliris is indicated in adults and children for the treatment of atypical haemolytic uremic syndrome (aHUS). | Nov. 29, 2023 | |
| See Detailed SMPC and OD Data | 224 | expired | Strensiq | asfotase alfa Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein | PLGB 31775/0004 | Strensiq is indicated for long-term enzyme replacement therapy in patients with paediatric-onset hypophosphatasia to treat the bone manifestations of the disease. | Sept. 1, 2025 | |
| See Detailed SMPC and OD Data | 225 | expired | Sylvant | siltuximab Chimeric-anti-interleukin-6 monoclonal antibody | PLGB 44185/0006 | Sylvant is indicated for the treatment of adult patients with multicentric Castleman’s disease (MCD) who are human immunodeficiency virus (HIV) negative and human herpesvirus 8 (HHV 8) negative. | May 27, 2024 | |
| See Detailed SMPC and OD Data | 226 | expired | TOBI Podhaler | tobramycin | PLGB 46302/0222 | TOBI Podhaler is indicated for the suppressive therapy of chronic pulmonary infection due to Pseudomonas aeruginosa in adults and children aged 6 years and older with cystic fibrosis. Consideration should be given to official guidance on the appropriate use of antibacterial agents. | July 25, 2023 | |
| See Detailed SMPC and OD Data | 227 | expired | Translarna | ataluren (3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid | PLGB 44221/0002 | Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing | Aug. 5, 2024 | |
| See Detailed SMPC and OD Data | 228 | expired | Vimizim | elosulfase alfa Recombinant human N-acetylgalactosamine-6-sulfatase | PLGB 45814/0007 | Vimizim is indicated for the treatment of mucopolysaccharidosis, type IVA (Morquio A Syndrome, MPS IVA) in patients of all ages. | April 30, 2024 | |
| See Detailed SMPC and OD Data | 229 | expired | VPRIV | velaglucerase alfa | PLGB 16189/0088 | VPRIV is indicated for long-term enzyme replacement therapy (ERT) in patients with type 1 Gaucher disease. | Aug. 30, 2022 | |
| See Detailed SMPC and OD Data | 230 | expired | Vyndaqel | tafamidis N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate | PLGB 00057/1660 | Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment. | Nov. 18, 2021 | |
| See Detailed SMPC and OD Data | 231 | expired | Wakix 4.5 mg Film-coated Tablets | PITOLISANT HYDROCHLORIDE | PLGB 18813/0007 | Wakix is indicated in adults for the treatment of narcolepsy with or without cataplexy | April 4, 2026 | |
| See Detailed SMPC and OD Data | 232 | expired | Wakix 18 mg Film-coated Tablets | PITOLISANT HYDROCHLORIDE | PLGB 18813/0006 | Wakix is indicated in adults for the treatment of narcolepsy with or without cataplexy | April 4, 2026 | |
| See Detailed SMPC and OD Data | 233 | expired | Welireg | BELZUTIFAN | PLGB 53095/0087 | Treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL-associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable or undesirable. | May 30, 2032 | |
| See Detailed SMPC and OD Data | 234 | expired | Xaluprine | mercaptopurine Mercaptopurine (oral suspension) | PLGB 13581/0002 | Treatment of acute lymphoblastic leukaemia (ALL) in adults, adolescents and children. | March 13, 2022 | |
| See Detailed SMPC and OD Data | 235 | expired | Zynteglo | betibeglogene autotemcel | PLGB 51370/0001 | Zynteglo is indicated for the treatment of patients 12 years and older with transfusion dependent ? thalassaemia (TDT) who do not have a ?0/?0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA) matched related HSC donor is not available | June 4, 2029 | |
| See Detailed SMPC and OD Data | 1 | current | ABECMA | idecabtagene vicleucel | PLGB 15105/0191 | Abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy | June 23, 2032 | |
| See Detailed SMPC and OD Data | 2 | current | Adzynma | recombinant human A disintegrin and metalloproteinase with thrombospondin motifs 13 (rADAMTS13) | PL 16189/0148 | Adzynma as an enzyme replacement therapy indicated for the treatment of ADAMTS13 deficiency in children and adult patients with congenital thrombotic thrombocytopenic purpura of all age groups | May 11, 2035 | |
| See Detailed SMPC and OD Data | 3 | current | AGAMREE | VAMOROLONE | PLGB 25835/0005 | Agamree in the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older. | Jan. 11, 2034 | |
| See Detailed SMPC and OD Data | 4 | current | Akantior | POLIHEXANIDE | PL 36078/0007 | Akantior is indicated for the treatment of acanthamoeba keratitis in adults and children from 12 years of age | May 15, 2035 | |
| See Detailed SMPC and OD Data | 5 | current | ALPROLIX | eftrenonacog alfa Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1 | PLGB 30941/0003 | Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency). ALPROLIX can be used for all age groups. | May 13, 2026 | |
| See Detailed SMPC and OD Data | 6 | current | ALYFTREK | TEZACAFTOR, DEUTIVACAFTOR, VANZACAFTOR CALCIUM DIHYDRATE | PL 22352/0024 | Alyftrek 50mg/20mg/4mg & 125mg/50mg/10mg film-coated tablets for the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. | March 7, 2035 | |
| See Detailed SMPC and OD Data | 7 | current | AMGLIDIA | Glibenclamide | PLGB 50687/0001 | AMGLIDIA is indicated for the treatment of neonatal diabetes mellitus, for use in newborns, infants and children. Sulphonylureas like AMGLIDIA have been shown to be effective in patients with mutations in the genes coding for the ?-cell ATP-sensitive potassium channel and chromosome 6q24-related transient neonatal diabetes mellitus. | May 28, 2028 | |
| See Detailed SMPC and OD Data | 8 | current | ARIKAYCE liposomal | Amikacin sulfate | PLGB 47434/0001 | ARIKAYCE liposomal is indicated for the treatment of non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium Complex (MAC) in adults with limited treatment options who do not have cystic fibrosis. | Oct. 28, 2030 | |
| See Detailed SMPC and OD Data | 9 | current | Artesunate Amivas | Artesunate | PLGB 55184/0002 | Artesunate Amivas is indicated for the initial treatment of severe malaria in adults and children | March 27, 2032 | |
| See Detailed SMPC and OD Data | 10 | current | ASPAVELI | pegcetacoplan | PLGB 30941/0022 | ASPAVELI is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months. | Feb. 24, 2032 | |
| See Detailed SMPC and OD Data | 11 | current | Aucatzyl | autologous T cells expressing an anti-CD19 chimeric antigen receptor. Autologous T cells that are genetically modified ex-vivo with a lentiviral vector to express a novel CD19 chimeric antigen receptor. | PLGB 46113/0001 | Aucatzyl is indicated for the treatment of adult patients (?18 years) with relapsed or refractory B cell precursor acute lymphoblastic leukaemia | April 25, 2035 | |
| See Detailed SMPC and OD Data | 12 | current | Avacopan Vifor | avacopan | PLGB 50784/0008 | Avacopan Vifor, in combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) | May 5, 2032 | |
| See Detailed SMPC and OD Data | 13 | current | Avacopan Vifor | avacopan | PLGB 50784/0008 | Avacopan Vifor, in combination with a rituximab or cyclophosphamide regimen, is indicated for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) | May 5, 2032 | |
| See Detailed SMPC and OD Data | 14 | current | AYVAKYT | avapritinib (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine | PLGB 52115/0001 | AYVAKYT is indicated as monotherapy for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumours (GIST) harbouring the platelet-derived growth factor receptor alpha (PDGFRA) D842V mutation | Sept. 25, 2030 | |
| See Detailed SMPC and OD Data | 15 | current | AYVAKYT | avapritinib (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine | PLGB 52115/0001 | AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN), or mast cell leukaemia (MCL). | Sept. 10, 2034 | |
| See Detailed SMPC and OD Data | 16 | current | AYVAKYT | avapritinib (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine | PLGB 52115/0006 | AYVAKYT is indicated as monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN), or mast cell leukaemia (MCL). | Sept. 10, 2034 | |
| See Detailed SMPC and OD Data | 17 | current | Besponsa | Inotuzumab ozogamicin | PLGB 00057/1546 | BESPONSA is indicated as monotherapy for the treatment of adults with relapsed or refractory CD22-positive B cell precursor acute lymphoblastic leukaemia (ALL). Adult patients with Philadelphia chromosome positive (Ph+) relapsed or refractory B cell precursor ALL should have failed treatment with at least 1 tyrosine kinase inhibitor (TKI). | July 3, 2027 | |
| See Detailed SMPC and OD Data | 18 | current | BLINCYTO | Blinatumomab | PLGB 13832/0018 | BLINCYTO is indicated as monotherapy for the treatment of adults with CD19 positive relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL). Patients with Philadelphia chromosome positive B-cell precursor ALL should have failed treatment with at least 2 tyrosine kinase inhibitors (TKIs) and have no alternative treatment options. BLINCYTO is indicated as monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive B-cell precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%. BLINCYTO is indicated as monotherapy for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19 positive B-cell precursor ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic haematopoietic stem cell transplantation. BLINCYTO is indicated for the treatment of adult patients with Philadelphia chromosome negative CD19-positive B-cell precursor leukaemia ALL in the consolidation phase. BLINCYTO is indicated for the treatment of paediatric patients aged 1 year or older with Philadelphia chromosome negative CD19-positive B-cell precursor leukaemia ALL in first relapse in the consolidation phase. | Nov. 25, 2027 | |
| See Detailed SMPC and OD Data | 19 | current | Brineura | cerliponase alfa Recombinant human tripeptidyl-peptidase 1 | PLGB 45814/0001 | Brineura is indicated for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency. | June 1, 2027 | |
| See Detailed SMPC and OD Data | 20 | current | Bylvay | Odevixibat sesquihydrate | PLGB 28247/0009 | Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. | Sept. 7, 2031 | |
| See Detailed SMPC and OD Data | 21 | current | Cablivi | caplacizumab Nanobody directed towards the human A1 domain of von Willebrand factor | PLGB 04425/0888 | Cablivi is indicated for the treatment of adults and adolescents of 12 years of age and older weighing at least 40 kg experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression. | Sept. 4, 2030 | |
| See Detailed SMPC and OD Data | 22 | current | Carvykti | Ciltacabtagene autoleucel | PLGB 00242/0745 | Treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide. | Jan. 4, 2033 | |
| See Detailed SMPC and OD Data | 23 | current | Casgevy | autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene | PLGB 22352/0019 | Authorised orphan indication: treatment of transfusion-dependent ?-thalassemia in patients 12 years of age and older for whom a human leukocyte antigen-matched related haematopoietic stem cell donor is appropriate and a human leukocyte antigen matched related haematopoietic stem cell donor is not available. | Nov. 15, 2033 | |
| See Detailed SMPC and OD Data | 24 | current | Casgevy | autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene | PLGB 22352/0019 | treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises who have the ?S/?S, ?S/?+ or ?S/?0 genotype, for whom a haematopoeitic stem cell transplantation is appropriate and a human leukocyte antigen matched related haematopoietic stem cell donor is not available. | Nov. 15, 2033 | |
| See Detailed SMPC and OD Data | 25 | current | Cerdelga | eliglustat | PLGB 04425/0763 | Cerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs). | Jan. 21, 2027 | |
| See Detailed SMPC and OD Data | 26 | current | Chenodeoxycholic acid Leadiant | Chenodeoxycholic acid | PLGB 44975/0001 | Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)) in infants, children and adolescents aged 1 month to 18 years and adults | April 12, 2027 | |
| See Detailed SMPC and OD Data | 27 | current | Coagadex | Human coagulation factor X | PLGB 08801/0059 | Coagadex is indicated for the treatment and prophylaxis of bleeding episodes and for perioperative management in patients with hereditary factor X deficiency. | March 18, 2028 | |
| See Detailed SMPC and OD Data | 28 | current | Cresemba | isavuconazole | PLGB 32205/0005 | Treatment of mucormycosis in patients from 1 year of age for whom amphotericin B is inappropriate. | Oct. 19, 2027 | |
| See Detailed SMPC and OD Data | 29 | current | Cresemba | isavuconazole | PLGB 32205/0005 | Treatment of invasive aspergillosis in patients from 1 year of age. | Oct. 19, 2027 | |
| See Detailed SMPC and OD Data | 30 | current | CRYSVITA | Burosumab Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 | PLGB 50262/0001 | CRYSVITA is indicated for the treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons | Feb. 21, 2028 | |
| See Detailed SMPC and OD Data | 31 | current | Cystadrops | mercaptamine Cysteamine hydrochloride | PLGB 15266/0021 | Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 2 years of age with cystinosis. | Jan. 23, 2027 | |
| See Detailed SMPC and OD Data | 32 | current | Darzalex | Daratumumab | PLGB 00242/0676 | • in combination with lenalidomide and dexamethasone or with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant • in combination with bortezomib, lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma* • in combination with bortezomib, thalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant • in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy • in combination with pomalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received one prior therapy containing a proteasome inhibitor and lenalidomide and were lenalidomide-refractory, or who have received at least two prior therapies that included lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or after the last therapy (see section 5.1)* • as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a proteasome inhibitor and an immunomodulatory agent and who have demonstrated disease progression on the last therapy • as monotherapy is indicated for the treatment of adult patients with smouldering multiple myeloma at high risk of developing multiple myeloma | May 24, 2026 | |
| See Detailed SMPC and OD Data | 33 | current | Darzalex | Daratumumab | PLGB 00242/0677 | Darzalex is indicated in combination with cyclophosphamide, bortezomib and dexamethasone for the treatment of adult patients with newly diagnosed systemic light chain (AL) amyloidosis | Oct. 11, 2031 | |
| See Detailed SMPC and OD Data | 34 | current | Daurismo | glasdegib maleate | PLGB 00057/1687 | Daurismo is indicated, in combination with low-dose cytarabine, for the treatment of newly diagnosed de novo or secondary acute myeloid leukaemia (AML) in adult patients who are not candidates for standard induction chemotherapy. | June 29, 2030 | |
| See Detailed SMPC and OD Data | 35 | current | Dovprela | pretomanid (S)-2-nitro-6-(4-(trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b][1,3]oxazine | PLGB 46302/0234 | Dovprela is indicated in combination with bedaquiline and linezolid, in adults, for the treatment of pulmonary extensively drug resistant (XDR), or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB). | Aug. 4, 2030 | |
| See Detailed SMPC and OD Data | 36 | current | Duvyzat | GIVINOSTAT HYDROCHLORIDE MONOHYDRATE | PLGB 13297/0017 | reatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. | Dec. 20, 2034 | |
| See Detailed SMPC and OD Data | 37 | current | Ebvallo 2.8 x 10^7 - 7.3 x 10^7 cells/mL dispersion for injection | TABELECLEUCEL | PLGB 00603/0249 | treatment of adult and paediatric patients 2 years of age and older with relapsed or refractory Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate’. | May 22, 2033 | |
| See Detailed SMPC and OD Data | 38 | current | Ekterly | Sebetralstat | PL4632 6/0001 | Ekterly is indicated for the treatment of hereditary angioedema (HAE) attacks in adult and adolescents aged 12 years and older. | July 15, 2035 | |
| See Detailed SMPC and OD Data | 39 | current | ELZONRIS | TAGRAXOFUSP | PLGB 53425/0001 | Monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm | Oct. 14, 2031 | |
| See Detailed SMPC and OD Data | 40 | current | Enspryng | Satralizumab | PLGB 00031/0916 | Enspryng is indicated as a monotherapy or in combination with immunosuppressive therapy (IST) for the treatment of neuromyelitis optica spectrum disorders (NMOSD) in adult and adolescent patients from 12 years of age who are anti-aquaporin-4 IgG (AQP4-IgG) seropositive. | Nov. 21, 2033 | |
| See Detailed SMPC and OD Data | 41 | current | Epidyolex | cannabidiol | PLGB 36772/0001 | Epidyolex is indicated for use as adjunctive therapy of seizures associated with Lennox Gastaut syndrome (LGS), in conjunction with clobazam, for patients 2 years of age and older. | Sept. 23, 2029 | |
| See Detailed SMPC and OD Data | 42 | current | Epidyolex | cannabidiol | PLGB 36772/0001 | Epidyolex is indicated for use as adjunctive therapy of seizures associated with Dravet syndrome (DS), in conjunction with clobazam, for patients 2 years of age and older. | Sept. 23, 2029 | |
| See Detailed SMPC and OD Data | 43 | current | Epidyolex | cannabidiol | PLGB 36772/0001 | Epidyolex is indicated for use as adjunctive therapy of seizures associated with tuberous sclerosis complex (TSC) for patients 2 years of age and older. | Aug. 5, 2031 | |
| See Detailed SMPC and OD Data | 44 | current | Ezmekly | mirdametinib | PL 59369/0003 | symptomatic, inoperable plexiform neurofibromas (PN) in paediatric and adult patients with NF1 aged 2 years and above | Dec. 11, 2035 | |
| See Detailed SMPC and OD Data | 45 | current | FABHALTA | IPTACOPAN HYDROCHLORIDE MONOHYDRATE | PLGB 00101/1231 | Monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia | Aug. 2, 2034 | |
| See Detailed SMPC and OD Data | 46 | current | FABHALTA | IPTACOPAN HYDROCHLORIDE MONOHYDRATE | PLGB 00101/1231 | treatment of adult patients with complement 3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, or in patients who are RAS-inhibitor intolerant, or for whom a RAS inhibitor is contraindicated | May 7, 2035 | |
| See Detailed SMPC and OD Data | 47 | current | Filspari | Sparsentan | PLGB 15240/0005 | Treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion > 1.0 g/day (or urine protein-to-creatinine ratio ? 0.75 g/g) | Nov. 6, 2034 | |
| See Detailed SMPC and OD Data | 48 | current | FILSUVEZ | BETULAE CORTEX DRY EXTRACT | PLGB 08829/0211 | Treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in patients 6 months and older | Aug. 11, 2032 | |
| See Detailed SMPC and OD Data | 49 | current | FINLEE | DABRAFENIB MESYLATE | PLGB 00101/1228 | Low-grade glioma Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy. High-grade glioma Finlee in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment. | March 20, 2036 | |
| See Detailed SMPC and OD Data | 50 | current | Fintepla | fenfluramine | PLGB 00039/0804 | Fintepla is indicated for the treatment of seizures associated with Dravet syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older. | Dec. 21, 2030 | |
| See Detailed SMPC and OD Data | 51 | current | Fintepla | fenfluramine hydrochloride | PLGB 00039/0804 | Treatment of seizures associated with Lennox- Gastaut syndrome as an add on therapy to other anti-epileptic medicines for patients 2 years of age and older. | July 5, 2033 | |
| See Detailed SMPC and OD Data | 52 | current | Galafold | migalastat | PLGB 25823/0002 | Galafold is indicated for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease (?-galactosidase A deficiency) and who have an amenable mutation (see the tables in section 5.1). | May 31, 2026 | |
| See Detailed SMPC and OD Data | 53 | current | Gilteritinib Astellas 40mg film-coated tablets | gilteritinib | PLGB 00166/0425 | Gilteritinib Astellas is indicated as monotherapy for the treatment of adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation. | Oct. 28, 2029 | |
| See Detailed SMPC and OD Data | 54 | current | Givlaari | givosiran | PLGB 50597/0004 | Treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older | March 4, 2030 | |
| See Detailed SMPC and OD Data | 55 | current | Hemgenix 1 x 10^13 genome copies/mL concentrate for solution for infusion | ETRANACOGENE DEZAPARVOVEC | PLGB 15036/0160 | treatment of severe and moderately severe Haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors. | March 22, 2033 | |
| See Detailed SMPC and OD Data | 56 | current | Holoclar | Ex-vivo expanded autologous human corneal epithelium containing stem cells | PLGB 58508/0001 | Treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity), unilateral or bilateral, due to physical or chemical ocular burns. A minimum of 1-2 mm2 of undamaged limbus is required for biopsy. | Feb. 19, 2027 | |
| See Detailed SMPC and OD Data | 57 | current | Hyftor | SIROLIMUS | PL 54373/0001 | Hyftor is indicated for the treatment of facial angiofibroma associated with tuberous sclerosis complex in adults and paediatric patients aged 6 years and older | Sept. 1, 2033 | |
| See Detailed SMPC and OD Data | 58 | current | Idefirix | imlifidase Recombinant IgG degrading enzyme of Streptococcus pyogenes | PLGB 46323/0002 | Idefirix is indicated for desensitisation treatment of highly sensitised adult kidney transplant patients with positive crossmatch against an available deceased donor. The use of Idefirix should be reserved for patients unlikely to be transplanted under the available kidney allocation system including prioritisation programmes for highly sensitised patients. | Sept. 1, 2030 | |
| See Detailed SMPC and OD Data | 59 | current | IDELVION | albutrepenonacog alfa Recombinant fusion protein linking human coagulation factor IX with human albumin | PLGB 15036/0141 | Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency). | May 13, 2026 | |
| See Detailed SMPC and OD Data | 60 | current | IDELVION | albutrepenonacog alfa Recombinant fusion protein linking human coagulation factor IX with human albumin | PLGB 15036/0155 | Treatment and prophylaxis of bleeding in patients with haemophilia B (congenital factor IX deficiency). | May 13, 2026 | |
| See Detailed SMPC and OD Data | 61 | current | Imcivree | setmelanotide | PLGB 55587/0001 | The treatment of obesity and the control of hunger associated with genetically confirmed loss of function biallelic pro-opiomelanocortin (POMC), including PCSK1 deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 2 years of age and above | Sept. 14, 2031 | |
| See Detailed SMPC and OD Data | 62 | current | Imcivree | setmelanotide | PLGB 55587/0001 | The treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) in adults and children 2 years of age and above | Nov. 17, 2032 | |
| See Detailed SMPC and OD Data | 63 | current | IMDYLLTRA 1 milligram and 10mg powder for concentrate and solution for infusion | TARLATAMAB | PLGB 13832/0083 | For the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after at least two prior lines of therapy including platinum-based chemotherapy | Dec. 31, 2034 | |
| See Detailed SMPC and OD Data | 64 | current | Inrebic | fedratinib dihydrochloride monohydrate | PLGB 15105/0177 | Inrebic is indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib. | April 16, 2031 | |
| See Detailed SMPC and OD Data | 65 | current | IQIRVO | Elafibranor | PLGB 34926/0029 | Iqirvo is indicated for the treatment of primary biliary cholangitis. | Oct. 1, 2034 | |
| See Detailed SMPC and OD Data | 66 | current | Isturisa | osilodrostat | PLGB 15266/0029 | Isturisa is indicated for the treatment of endogenous Cushing’s syndrome in adults. | Jan. 13, 2030 |