Posted:
The FDA approved Otarmeni (lunsotogene parvec-cwha) on April 23, 2026, the first gene therapy indicated for hearing loss caused by variants in the OTOF gene. The therapy is made by Regeneron Pharmaceuticals.
Otarmeni is approved for pediatric and adult patients with severe-to-profound sensorineural hearing loss associated with confirmed biallelic variants in the OTOF gene. Patients must have preserved outer hair cell function and no prior cochlear implant in the same ear. The OTOF gene is responsible for producing otoferlin, a protein needed to transmit sound signals from the inner ear to the brain.
The therapy is delivered as a single surgical dose into the cochlea using a dual adeno-associated virus vector. It works by introducing a functional copy of the OTOF gene into inner hair cells to restore otoferlin production.
The FDA based its approval on an ongoing clinical trial of 24 pediatric patients between 10 months and 16 years of age. Of the 20 patients evaluable for efficacy, 80% showed improved hearing, an outcome the FDA notes is not expected in the natural history of the condition without treatment.
The approval is accelerated. Continued approval may depend on confirmatory data assessing durability of hearing improvement and effects on speech development and quality of life. Common side effects included middle ear infection, nausea, dizziness, and procedural pain. The therapy is not recommended for patients whose anatomy would prevent safe access to the inner ear.
Learn more here.
