The U.S. Food and Drug Administration (FDA) has approved the first-ever gene therapy for inherited deafness. The therapy, named Otarmeni, targets a form of hearing loss caused by mutations in the OTOF gene, which is crucial for transmitting sound signals from the inner ear to the brain. When this gene is defective, it results in severe hearing loss.
Clinical trials showed significant improvements, with some participants able to hear whispers and others achieving a level of hearing that negates the need for cochlear implants. The therapy, developed by Regeneron, is approved for both children and adults with OTOF-related hearing loss. Regeneron has stated the treatment will be free to U.S. patients, excluding administration costs.
Experts hail this as a landmark moment, accelerating the development of future genetic therapies for hearing loss. The approval process was notably rapid, facilitated by the FDA's "fast track" designation due to the lack of existing treatments addressing the root cause of this specific hearing loss. OTOF-related hearing loss affects approximately 50 newborns annually in the U.S.