FDA Policy/General

Gene Therapy Approved for Inherited Hearing Loss

FDA has approved a Regeneron Pharmaceuticals BLA for Otarmeni (lunsotogene parvec-cwha), the first gene therapy for a form of inherited hearing loss. The dual adeno-associated virus (AAV) vector-based gene therapy is indicated for patients with severe-to-profound sensorineural hearing loss caused by mutations in the OTOF gene.

The approval came just 61 days after the biologics license application was filed, making it one of the fastest reviews in modern FDA history and the first gene therapy authorized under the Commissioner’s National Priority Voucher pilot program.

Hearing loss linked to OTOF mutations accounts for a small but meaningful share of inherited, non-syndromic deafness, according to FDA. Patients with the condition lack functional otoferlin, a protein critical for transmitting sound signals from the inner ear to the brain. Until now, no disease-modifying treatments were available.

Otarmeni works by delivering a functional copy of the OTOF gene directly into inner ear cells using a dual AAV vector system, the agency says. The therapy is administered as a one-time surgical procedure, with the gene delivered into the cochlea via a specialized infusion system.

The approval was based on an ongoing clinical trial involving 24 pediatric patients. Among 20 evaluable participants, 80% showed improvements in hearing — an outcome not typically seen in the natural progression of the disease, FDA says. The study also demonstrated sustained production of otoferlin following treatment.

Side effects reported in the trial included middle ear infections, dizziness, nausea, and procedural pain. The therapy is not recommended for patients whose anatomy prevents safe access to the inner ear.

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