Regeneron’s new gene therapy, Otarmeni, received FDA approval on April 23, 2026. The dual adeno‑associated virus (AAV) vector targets the OTOF gene, responsible for a subset of congenital deafness. With a single cochlear injection, the therapy delivers a functional gene copy to inner hair cells, aiming to restore auditory signaling for patients who have not yet received a cochlear implant.

The 61‑day review, the fastest in modern FDA history, followed a New England Journal of Medicine study showing 80% of 20 evaluable patients gained hearing improvements. The therapy earned orphan drug, rare pediatric disease, fast‑track, and RMAT designations, underscoring its potential to fill a long‑standing unmet need in genetic hearing loss for children and adults who are otherwise untreated.

Otarmeni combines a biologic and a device: a syringe, catheter, and infusion pump deliver the dual‑vector therapy directly into the cochlea. Side effects include middle‑ear infection, nausea, dizziness, and procedural pain, while surgical complications must be monitored. The FDA’s approval marks a tangible step toward treating inherited deafness, offering a new option for the affected population.