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Orchard Therapeutics Submits BLA to FDA for Early-Onset Metachromatic Leukodystrophy Gene Therapy

NEW YORK – Gene therapy developer Orchard Therapeutics on Thursday said it has completed its rolling biologics license application (BLA) submission to the US Food and Drug Administration for OTL-200, the company's metachromatic leukodystrophy candidate.

OTL-200, an investigational ex vivo autologous hematopoietic stem cell gene therapy for certain patients with early-onset metachromatic leukodystrophy, delivers a functional copy of the ARSA gene. Mutations in ARSA cause the rare genetic disorder, in which sulfatides accumulate in cells. 

The gene therapy is already approved by the European Commission and UK Medicines and Healthcare Products Regulatory Agency, where it is branded Libmeldy (atidarsagene autotemcel). In the second quarter of 2023, Libmeldy brought in $6.7 million in revenue, a 76 percent increase from $3.8 million in the year-ago quarter. Libmeldy sales comprised the majority of the company's overall Q2 revenues of $7.3 million.

London-based Orchard said it expects to hear whether the FDA has accepted its BLA in the third quarter. Orchard requested priority review from the agency, which, if granted, puts the company on track to receive a regulatory decision in the first half of next year. The agency previously granted OTL-200 rare pediatric disease and regenerative medicine advanced therapy designations.

In June, Orchard raised $34 million in a second closing of a securities purchase agreement, which is part of a larger financing plan that could bring in up to $188 million at increasing valuations if OTL-200 achieves certain US regulatory milestones.

That additional funding "ensures we are well-capitalized to progress US launch preparations [for OTL-200], continue investing in initiatives aimed at accelerating commercial growth in Europe, and advance our next-in-line neurometabolic programs in [mucopolysaccharidoses] disorders," Orchard CEO Bobby Gaspar said in a statement. "The next 12 months have the potential to provide Orchard Therapeutics several breakout opportunities as we work to cement our leadership position in the [hematopoietic stem cell] gene therapy field."

As of June 30, Orchard had $38.2 million in cash and cash equivalents and $112.5 million in marketable securities.