Orchard Therapeutics Says FDA Accepts BLA For OTL-200 In Metachromatic Leukodystrophy

(RTTNews) - Orchard Therapeutics (ORTX), a global gene therapy leader, announced Monday that the U.S. Food and Drug Administration (FDA) has accepted the filing of its Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review.

The BLA for OTL-200 is based on data from 39 pediatric patients with early-onset MLD, enrolled in two prospective non-randomized clinical studies or treated under expanded access frameworks, who were administered OTL-200 and compared with natural history data from 49 untreated patients.

In clinical trials, treatment with OTL-200 resulted in preservation of motor function and cognitive development in most patients compared to disease natural history with up to 12 years of follow-up.

The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.

OTL-200 would be the first and only treatment in the U.S. for early-onset MLD. The company said it is working diligently in parallel to prepare for a potential launch in 2024.

The previously published data demonstrated administration of one-time gene therapy enables sustained preservation of motor function and cognitive development.

OTL-200 previously received both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA.