FDA grants accelerated approval for Regeneron’s Otarmeni

Regeneron (REGN) Pharmaceuticals announced the U.S. Food and Drug Administration has granted accelerated approval for Otarmeni, the first gene therapy and second new molecular entity approved under the FDA Commissioner’s National Priority Voucher program. Otarmeni is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. Otarmeni, formerly known as DB-OTO, is the first and only in vivo gene therapy for OTOF-related hearing loss and will be made available by Regeneron for free in the U.S. Otarmeni was granted accelerated approval based on the improvement of hearing sensitivity by average pure tone audiometry at week 24. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory portion of the CHORD clinical trial. Otarmeni is not recommended in patients in whom preoperative imaging demonstrates that access to the inner ear is not feasible, including those with abnormal mastoid pneumatization or clinically significant anatomic variations of the middle ear and inner ear. “The FDA approval of Otarmeni signals a new era in the treatment of genetic forms of hearing loss, where reinstating 24/7 natural hearing is now possible,” said A. Eliot Shearer, M.D., Ph.D., otolaryngologist in the department of Otolaryngology and Communication Enhancement at Boston Children’s Hospital, Associate Professor of Otolaryngology-Head and Neck Surgery at Harvard Medical School and a CHORD trial investigator. “In the pivotal trial, the one-time gene therapy demonstrated rapid, meaningful and consistent hearing responses, with most children achieving remarkable hearing improvements. I’ve witnessed firsthand my trial participant responding to their mother’s voice, dancing to music and interacting with the world, and these moments are now possible for more children born with this specific form of hearing loss.”