FDA Accepts Orchard Therapeutics’ Biologics License Application, PDUFA Date Set

Global gene therapy leader, Orchard Therapeutics (Nasdaq: ORTX), has submitted a Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review. The FDA has accepted Orchard’s BLA and has set a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.

Bobby Gaspar, M.D., Ph.D., co-founder and chief executive officer of Orchard Therapeutics, said, “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.”


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.

Orchard’s Libmeldy (OTL-200) is the first therapy approved for eligible patients with early-onset MLD. Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the U.S.

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

MLD is a rare and life-threatening inherited disease of the body’s metabolic system that results in nervous system damage, and patients with MLD gradually lose the ability to move, talk, swallow, eat and see.

About Orchard Therapeutics

At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard is advancing a pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit www.orchard-tx.com, and follow us on X (Twitter) and LinkedIn.

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FDA Accepts Orchard Therapeutics’ Biologics License Application, PDUFA Date Set

Catie Corcoran

Biotech Editor