ORTX - FDA signs-off Orchard Therapeutics' gene therapy for rare nerve cell disorder

The FDA has signed-off Orchard Therapeutics' ([[ORTX]] +3.2%) Investigational New Drug application for OTL-200, an autologous, hematopoietic stem cell, lentiviral vector-based gene therapy in development for the treatment of metachromatic leukodystrophy ((MLD)). The company also has applied for Regenerative Medicine Advanced Therapy designation for OTL-200 to help facilitate additional dialogue with the FDA.As part of the IND filing, Orchard provided to the FDA data on 39 patients, including 9 patients from the U.S., who have received OTL-200 as part of clinical studies and compassionate use programs conducted at the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy. The company has post-treatment follow-up data of up to eight years in the earliest treated patients in these programs.Last month, the company received positive CHMP opinion recommending approval of Libmeldy (OTL-200).

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FDA signs-off Orchard Therapeutics' gene therapy for rare nerve cell disorder