ORTX - Orchard Therapeutics' OTL-200 wins FDA's RMAT status

The FDA has granted Orchard Therapeutics' (ORTX) OTL-200 its Regenerative Medicine Advanced Therapy ((RMAT)) designation for the treatment of early-onset metachromatic leukodystrophy ((MLD)), a rare hereditary disorder characterised by accumulation of fats called sulfatides.The agency's RMAT designation program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure a serious condition.In late 2020, the FDA cleared the company’s Investigational New Drug ((IND)) application for OTL-200, and the therapy also recently was approved in the European Union ((EU)) under the brand name, Libmeldy.Orchard shares up ~7% premarket.

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Orchard Therapeutics' OTL-200 wins FDA's RMAT status