Overview
- FDA regulators granted accelerated approval Thursday under the National Priority Voucher program after a 61‑day review, with continued approval contingent on confirming lasting clinical benefit.
- In the CHORD study, 16 of 20 participants showed hearing gains by 24 weeks, and five of 12 followed to 48 weeks reached near‑normal hearing that included whispers.
- Eligibility requires molecularly confirmed biallelic OTOF variants, severe‑to‑profound sensorineural hearing loss, preserved outer hair cell function, and no prior cochlear implant in the ear to be treated.
- The one‑time treatment is delivered by a trained surgeon as an intracochlear infusion using a cochlear implant–like approach, and common side effects were surgery‑related such as ear infections, nausea, dizziness, and procedural pain.
- Regeneron will provide Otarmeni at no cost to eligible U.S. patients, though families may still face facility or surgical charges until coverage is set.