Delandistrogene moxeparvovec-rokl
Orphan Drug Cold Chain RequiredFDA Approved
Description
Delandistrogene moxeparvovec-rokl is an adeno-associated virus gene therapy approved for Duchenne muscular dystrophy. It is one of the most visible recent rare-disease launches because it combines a one-time treatment model with complex administration and site-of-care logistics. Access is tightly linked to specialized neuromuscular centers and gene-therapy infrastructure.
Indications & Therapeutic Use
Duchenne muscular dystrophy
Linked Diseases:
Duchenne Muscular Dystrophy
G71.01D020388
Global Availability (1 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — |
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Delandistrogene moxeparvovec-rokl
| Generic Name | delandistrogene moxeparvovec-rokl |
| Brands | 1 brand available |
| Active Ingredient | delandistrogene moxeparvovec-rokl |
| Drug Class | Duchenne muscular dystrophy |
| Manufacturer | Sarepta Therapeutics |
| Dosage Forms | Concentrate for solution for infusion |
| Medical Code | M09AX |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT03375164 |
| Countries | 1 countries |
Authority Signals
Last Verified7/24/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations2 Validated Nodes