NEW YORK – The US Food and Drug Administration has accepted the biologics license application (BLA) for Orchard Therapeutics' metachromatic leukodystrophy gene therapy candidate and granted the drug priority review, the firm said Monday.
The FDA expects to issue its decision on OTL-200, for which London-based Orchard submitted a BLA in August, by March 18.
If approved, the gene therapy would be the first treatment greenlighted by the FDA for early-onset metachromatic leukodystrophy. The drug is already approved by the European Commission and UK Medicines and Healthcare Products Regulatory Agency, where it is branded Libmeldy (atidarsagene autotemcel).
OTL-200 is an investigational ex vivo autologous hematopoietic stem cell gene therapy for certain patients with early-onset metachromatic leukodystrophy, a rare genetic disorder caused by mutations in the ARSA gene that lead sulfatides to accumulate in cells. The one-time gene therapy is designed to deliver a functional copy of the ARSA gene.
The BLA is based on data from 39 patients in two prospective non-randomized clinical studies and patients treated under an expanded access framework who were administered the gene therapy and compared to a natural history cohort of 49 patients who did not receive treatment. Most patients treated with OTL-200 experienced preservation of motor function and cognitive development and had no treatment-related serious adverse events or deaths.
"We look forward to collaborating with the FDA throughout the review and evaluation of our application," said Orchard Cofounder and CEO Bobby Gaspar in a statement. "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."
The FDA previously granted OTL-200 rare pediatric disease and regenerative medicine advanced therapy designations.