Orchard Therapeutics announces FDA accepted BLA for OTL-200 in MLD

Orchard Therapeutics announced 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 agency has set a Prescription Drug User Fee Act, PDUFA, goal date of March 18, 2024. “Today is another significant step forward for patients and families in the U.S. impacted by this devastating and cruel disease who for too long have dealt with the unimaginable burden of going through the diagnostic odyssey, being told there were no treatments beyond supportive care, and then having to watch their child slip away,” said Bobby Gaspar, M.D., Ph.D., co-founder and chief executive officer of Orchard Therapeutics. “We look forward to collaborating with the FDA throughout the review and evaluation of our application. Due to the nature of the disease and the urgency to treat children affected by MLD, we are working diligently in parallel to prepare for a potential launch in 2024 and ensure OTL-200 will be available to patients in the U.S. as quickly as possible.”