Detailed SMPC and OD register data for: Libmeldy
Data as extracted from Orphan Designation Register
Product (OD)
Libmeldy
PL number (OD)
PLGB 49055/0002
OD Designation ID (OD)
OD1
Active substance (OD)
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
Data as extracted from SMPC. (PS: More information at bottom of the page)
Product (SMPC)
PL Number (SMPC)
Composition/ Active Substance (SMPC)
Dose form (SMPC)
Marketing Authorisation holder details (SMPC)
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SMPC composition / active substance
Matched EMA substance
EMA SMS ID
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Further information for:
Select a section below to read the detailed content extracted from the SMPC / Orphan Designation register.
OD Indication (OD)
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
Full Designation Number(OD)
PLGB 49055/0002/OD1
Orphan Condition(OD)
Metachromatic leukodystrophy
All Indications (SMPC)
All Contraindications (SMPC)
Warnings/Precautions (SMPC)
Interactions (SMPC)
Pregnancy/Lactation (SMPC)
Driving/Machines (SMPC)
Undesirable effects (SMPC)
Overdose (SMPC)
Shelf life (SMPC)
Storage (SMPC)
Container description (SMPC)
Handling/Disposal (SMPC)
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