FDA Approves First Gene Therapy for OTOF Deafness

The U.S. Food and Drug Administration approved Regeneron's Otarmeni on Thursday, marking the first gene therapy for inherited hearing loss caused by mutations in the OTOF gene. The one-time treatment targets a rare form of congenital deafness that affects approximately 50 newborns annually in the United States, restoring the body's ability to produce otoferlin, a protein essential for transmitting sound signals from inner ear cells to the brain.

Otarmeni uses a harmless virus to deliver a functional copy of the OTOF gene directly into the inner ear through a surgical procedure similar to cochlear implant insertion, performed under general anesthesia. Without otoferlin, individuals experience severe to profound hearing loss from birth, as sound vibrations fail to reach the auditory nerve. Prior to this therapy, the only option was cochlear implants, which provide partial hearing restoration but require ongoing maintenance and do not fully replicate natural sound fidelity.

The approval stems from a clinical trial involving 20 children and adults with confirmed OTOF mutations and no prior cochlear implants in the treated ear. Results showed hearing improvements in 16 participants...