Atidarsagene autotemcel
Approved · EMAAlso known as Autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene, Autologous CD34+ cells transduced with lentiviral vector which encodes for the aryl Sulfatase A complimentary deoxyribonucleic acid sequence, Lenmeldy.
Recent clinical, regulatory, research and industry developments relating to this drug.
Profile
Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.
Regulatory timeline
The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.
Indication: Libmeldy 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… Show full indicationShow less
Libmeldy 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.
Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.
- RxNorm (U.S. National Library of Medicine) — drug identity
- Regulatory event sources are credited in the Regulatory Timeline above.