Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S.
The FDA has granted accelerated approval to Otarmeni, a gene therapy for severe-to-profound and profound sensorineural hearing loss associated with biallelic variants in the OTOF gene. Regeneron will provide Otarmeni for free in the U.S., with 80% of CHORD trial participants achieving or surpassing the primary endpoint.
Regeneron Pharmaceuticals announced that the FDA has granted accelerated approval for Otarmeni, a gene therapy for pediatric and adult patients with severe-to-profound sensorineural hearing loss associated with biallelic variants in the OTOF gene. Otarmeni is an adeno-associated virus vector-based gene therapy. The approval is based on the CHORD trial, where 80% of participants achieved or surpassed the primary endpoint. Regeneron will provide Otarmeni for free in the U.S. OTOF-related hearing loss affects about 50 newborns per year in the U.S. Continued approval may be contingent upon verification of clinical benefit in the confirmatory portion of the CHORD trial.
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