FDA Approves Regeneron’s First-in-Class Gene Therapy for OTOF Hearing Loss
Regeneron Pharmaceuticals has received FDA accelerated approval (lunsotogene parvec-cwha), a gene therapy for patients with severe-to-profound sensorineural hearing loss caused by OTOF gene mutations.
The therapy is the first gene therapy approved under the FDA Commissioner’s National Priority Voucher programme and the first in vivo treatment for OTOF-related hearing loss.
Otarmeni is designed for paediatric and adult patients with confirmed biallelic OTOF variants, preserved outer hair cell function and no prior cochlear implant in the same ear. It will be made available free of charge to eligible patients in the United States.
Approval is based on data from the CHORD trial, where 80% of participants showed improved hearing sensitivity at 24 weeks, while 70% demonstrated improved auditory brainstem responses. Benefits were sustained in patients followed up to 48 weeks.
OTOF-related hearing loss is an ultra-rare condition affecting around 50 newborns annually in the US. Otarmeni is delivered through a single intracochlear infusion and must be administered by trained surgeons.
Common side effects include middle ear infection, dizziness and nausea. Continued approval is subject to confirmation of clinical benefit in ongoing studies.
