Biotechnology · us
U.S. Approves First Gene Therapy for Genetic Hearing Loss, Otarmeni Targets Rare OTOF Mutations
Regeneron’s Otarmeni has received FDA accelerated approval for certain patients with OTOF-related severe to profound sensorineural hearing loss; early trial data showed hearing improvement in most participants, but continued approval still requires further verification of clinical benefit.
The U.S. Food and Drug Administration has granted accelerated approval to Regeneron’s Otarmeni (lunsotogene parvec-cwha), making it the first gene therapy approved in the United States for genetic hearing loss. The approval is targeted at patients with severe to profound sensorineural hearing loss caused by biallelic variants in the OTOF gene who still retain outer hair cell function.
The OTOF gene is responsible for producing the otoferlin protein, which is important for signal transmission between sensory cells in the inner ear and the auditory nerve. When genetic variants leave the protein with insufficient function, the ear’s structure may remain relatively intact, but sound signals cannot be effectively transmitted to the nervous system, leading to severe hearing loss in early infancy or childhood.
Otarmeni is an in vivo gene therapy delivered using an adeno-associated viral vector and designed to deliver a functional OTOF gene into the cochlea. The drug must be administered through intracochlear infusion, a procedure with similarities to cochlear implant surgery, so it is not a standard outpatient injectable treatment and requires a surgical team with relevant experience and training.
The approval is based mainly on data from the CHORD trial. Regeneron said that among 20 treated participants aged 10 months to 16 years, 16 met the primary hearing improvement endpoint at week 24 as assessed by pure-tone audiometry; other participants reached the normal hearing range after longer follow-up. These results extend gene therapy beyond its traditionally more common use in blood, immune, or metabolic diseases and further into the field of sensory function restoration.
However, this is still an accelerated approval, not a complete final answer. CHORD is an ongoing phase 1/2, multicenter, open-label trial with a limited sample size, and the currently public information comes mainly from company press releases. Whether the FDA maintains this indication may depend on whether confirmatory trials can further verify clinical benefit and long-term safety.
Safety also needs to be placed on equal footing. Common adverse reactions listed in company data include otitis media, vomiting, nausea, dizziness, procedural pain, abnormal gait, and nystagmus; risks related to inner-ear surgery also include infection, cerebrospinal fluid leakage, vertigo, or other complications. For young children and families, treatment choices still need to be evaluated with help from the medical team alongside options such as cochlear implants, hearing-assistive devices, speech and sign-language support.
Regeneron said Otarmeni will be available in the United States to clinically eligible patients at no drug cost, but the practical burdens of surgery, care, and insurance reimbursement may not therefore disappear entirely. The scientific significance of this approval is clear: for the first time, it gives certain forms of genetic hearing loss an approved therapy that repairs the molecular defect; its public health and real-world clinical impact will still depend on access to diagnosis, long-term follow-up data, and whether health care systems can support its delivery.