Rolling submission of OTL 200 to FDA completed for metachromatic leukodystrophy.- Orchard Therapeutics

OTL 200 previously received both Rare Pediatric Disease (RPD) and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Orchard Therapeutics has requested priority review, which if granted, would put OTL 200 on track for a potential U.S. approval in the first half of 2024.

The most common adverse reaction attributed to treatment with OTL 200 was the occurrence of anti-ARSA antibodies. In addition to the risks associated with the gene therapy, treatment with OTL 200 is preceded by other medical interventions, namely bone marrow harvest or peripheral blood mobilization and apheresis, followed by myeloablative conditioning, which carry their own risks. During the clinical studies of OTL 200, the safety profiles of these interventions were consistent with their known safety and tolerability.