Atidarsagene autotemcel
Orphan Drug Cold Chain RequiredEMA Approved
Contents
Description
Libmeldy is an autologous hematopoietic stem cell gene therapy for metachromatic leukodystrophy. It involves transduction of patient CD34+ cells with a lentiviral vector encoding functional arylsulfatase A enzyme. The therapy is approved for pre-symptomatic late infantile and early juvenile forms, with ongoing studies in adult-onset disease.
Indications & Therapeutic Use
metachromatic leukodystrophy
Linked Diseases:
Global Availability (6 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Atidarsagene autotemcel
| Generic Name | atidarsagene autotemcel |
| Brands | 1 brand available |
| Active Ingredient | autologous CD34+ cells encoding arylsulfatase A gene |
| Drug Class | metachromatic leukodystrophy |
| Manufacturer | Orchard Therapeutics |
| Dosage Forms | IV infusion, cell suspension |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 90 days |
| Reg. Status | EMA Approved |
| Clinical Trial | NCT01560182 |
| Countries | 6 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations7 Validated Nodes