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. Due to the nature of the disease and the urgency to treat children affected by MLD, the company 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.

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 (n=30) or treated under expanded access frameworks (n=9), who were administered OTL-200 and compared with natural history data from 49 untreated patients. All treated patients were administered OTL- 200 and subsequently monitored at Ospedale San Raffaele in Milan, Italy. With more than a cumulative 250 patient-years of follow-up, treatment with OTL-200 was generally well-tolerated, with no treatment-related serious adverse events or deaths.

Most adverse events were associated with busulfan conditioning or background disease. The full clinical results comprising the BLA dataset were recently presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2023 in Jerusalem. MLD is a rare and life-threatening inherited disease of the body estimated to occur in approximately one in every 100,000 live births based on existing literature.

MLD is caused by a mutation in thearylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. During the clinical studies of Libmeldy, the safety profiles of these interventions were consistent with their known safety and tolerability. For more information about Libmeldy, please see the Summary of Product Characteristics (SmPC) available on the EEMA website.

Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchardardsardsardsards, the company is working diligently in parallel to collaborating with the FDA throughout the review and evaluation of application.

Due to the nature of The disease and the urgency to treating children affected by MLD, company are working diligently in parallelto prepare for a potential launch in 2022 and ensure OTL-200 would be available to patients in the US. as quickly as possible.