Regeneron Pharmaceuticals Inc. (NASDAQ: REGN) received U.S. Food and Drug Administration accelerated approval for Otarmeni, a gene therapy for treating severe-to-profound sensorineural hearing loss caused by OTOF gene variants.

The therapy targets an ultra-rare condition affecting approximately 50 newborns annually in the United States. Patients must have molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the treated ear.

The approval stems from the CHORD clinical trial involving 20 participants aged 10 months to 16 years. Results showed 80% of participants experienced hearing improvements to 70 decibels or better at 24 weeks, meeting the primary endpoint. Additionally, 70% demonstrated auditory brainstem response at 90 decibels or better.

Among participants followed to 48 weeks, all prior responders maintained their response, and 42% achieved normal hearing including whisper detection at 25 decibels or better.

The most common adverse reactions included otitis media, vomiting, nausea, dizziness, procedural pain, gait disturbance, and nystagmus. The therapy requires administration by surgeons experienced in intracochlear surgery using a procedure similar to cochlear implantation.

Regeneron stated it will provide Otarmeni at no cost to clinically eligible individuals in the United States, though administration costs may apply separately. The company plans regulatory submissions in additional markets.

The FDA granted Otarmeni multiple designations including Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy. The European Medicines Agency also granted Orphan Drug Designation.

Continued approval may depend on verification of clinical benefit in the confirmatory portion of the CHORD trial, according to the press release.