Regeneron Breaks the Silence with Otarmeni, a First-in-Class Hearing Loss Gene Therapy, Now FDA Approved

The U.S. Food and Drug Administration has granted accelerated approval to Otarmeni™ (lunsotogene parvec-cwha), a gene therapy developed by Regeneron Pharmaceuticals for pediatric and adult patients with severe-to-profound sensorineural hearing loss caused by biallelic mutations in the OTOF gene.

The therapy is the first in vivo gene therapy for this condition and was cleared under the FDA Commissioner’s National Priority Voucher program. It is indicated for patients with hearing thresholds greater than 90 dB hearing level who have preserved outer hair cell function and no prior cochlear implant in the same ear.

Delivered as a one-time intracochlear infusion using an adeno-associated virus vector, the therapy introduces a functional copy of the OTOF gene to restore production of otoferlin, a protein essential for transmitting auditory signals from inner ear sensory cells to the auditory nerve.

The approval is based on 24-week data from the ongoing Phase 1/2 CHORD trial, (NCT05788536) where 80% of participants achieved hearing thresholds of ≤70 dB HL, meeting the primary endpoint, and 70% demonstrated auditory brainstem responses at ≤90 dB. Among patients followed to 48 weeks, all responders maintained benefit, and 42% achieved hearing levels within the normal range, including the ability to detect whispers. Continued approval is contingent on confirmatory data from the trial.

Eliot Shearer MD PhD, CHORD trial investigator described the approval as a significant shift in treating genetic hearing loss, citing rapid, consistent, and meaningful improvements observed in the trial, with many children showing functional gains such as responding to sound and interacting more actively with their environment.

George D. Yancopoulos M.D., Ph.D. President and Chief Scientific Officer of Regeneron characterized the therapy as a major scientific advancement, highlighting its transformative impact in clinical studies and the company’s decision to provide the treatment at no cost to eligible patients in the United States.

The most common adverse events included otitis media, nausea, vomiting, dizziness, procedural pain, gait disturbance, and nystagmus. Administration requires a surgical procedure similar to cochlear implantation and must be performed by trained surgeons.

As part of a separate agreement with the U.S. government, Regeneron stated that it will provide Otarmeni free of cost to eligible patients in the United States, aiming to expand access for individuals with severe to profound hearing loss caused by OTOF gene variants.

In a statement, Yancopoulos noted that offering the therapy at no cost reflects the company’s commitment to advancing innovative treatments while improving patient access.

Otarmeni has received multiple regulatory designations, including Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy status, and further regulatory submissions are planned in additional markets as the CHORD trial continues to evaluate long-term safety and efficacy.

Reference

Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S. | Regeneron Pharmaceuticals Inc.

Regeneron Announces Agreement with U.S. Government to Help Lower Drug Costs for American Patients and Will Provide Innovative New Gene Therapy for Free in the U.S. | Regeneron Pharmaceuticals Inc.

A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/​Infants with Hearing Loss Due to Otoferlin Mutations (CHORD), ClinicalTrials.gov ID NCT05788536, https://clinicaltrials.gov/study/NCT05788536