FDA Approves First Gene Therapy for Hearing Loss

The first step has been taken toward treating genetic hearing loss. Otarimeni is the first gene therapy approved through the FDA's CNPV pilot program. In a statement, FDA Commissioner Marty Makary said, "This approval is a significant milestone in the treatment of genetic hearing loss."

Treatment efficacy was confirmed through clinical trial results published in the 2025 edition of The New England Journal of Medicine. In the study, the therapeutic agent was administered to 12 patients with hearing loss associated with the OTOF gene, resulting in hearing improvement in three individuals.

Otarimeni is administered through a one-time surgery. It involves injecting the drug directly into the inner ear (cochlea) of both ears using a needle and a fine tube. The therapeutic agent delivers a normal copy of the OTOF gene to key cells in the inner ear, restoring the function of the protein otoperrlin, which is necessary for transmitting auditory signals to the brain.

However, it cannot be applied to ears that have undergone cochlear implantation, and its use is recommended only for patients whose outer hair cell function is preserved.