FDA approves gene therapy Otarmeni for OTOF hearing loss

The FDA approved lunsotogene parvec-cwha (Otarmeni, Regeneron), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy. Otarmeni is indicated for pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic variants in the OTOF (otoferlin) gene with preserved outer hair cell function and no prior cochlear implant in the same ear.

Otarmeni and the administration kit are a one-time biologic-device combination product. The therapy delivers a functional copy of the OTOF gene to inner hair cells to restore otoferlin production and auditory signaling. Common side effects included middle ear infection, nausea, dizziness, and procedural pain.

The approval was supported by data from an ongoing multicenter, single-arm trial that enrolled 24 pediatric patients ages 10 months to 16 years. Among the 20 patients evaluable for efficacy, 80% experienced improvements in hearing.

Following the publication of hearing restoration results in the New England Journal of Medicine, the FDA granted a national priority voucher for accelerated review. The approval came 61 days after BLA filing, making it the sixth approval under the Commissioner’s National Priority Voucher pilot program and the first gene therapy product approved under the program. Continued approval may be contingent upon assessment of durability of hearing improvement and verification of treatment effects on speech development and quality of life.

Source(s): FDA press announcement (April 23, 2026): https://www.fda.gov/news-events/press-announcements/fda-approves-first-ever-gene-therapy-treatment-genetic-hearing-loss-under-national-priority-voucher