FDA approves Otarmeni the first gene therapy for genetic hearing loss as Regeneron commits to offering it for free

Regeneron’s Otarmeni (lunsotogene parvec-cwha) has recently received FDA approval as the first ever gene therapy for genetic hearing loss, and the company has committed to making it available at no cost to eligible patients in the United States.

The therapy is indicated for pediatric and adult patients with severe to profound sensorineural hearing loss associated with biallelic variants in the OTOF gene. OTOF-related hearing loss is an ultra-rare condition affecting around 50 newborns per year in the U.S. Though all structures within the ear remain intact, variants in the OTOF gene prevent production of functional otoferlin protein, which is critical for communication between the inner ear’s sensory cells and the auditory nerve. Until now, the condition was considered permanent and managed with lifelong use of devices.

Approval was based on results from the pivotal CHORD trial, in which 20 participants aged 10 months to 16 years received a single dose via intracochlear infusion. Eighty percent of participants achieved meaningful hearing improvements at 24 weeks, meeting the trial’s primary endpoint. Among those followed to 48 weeks, 42% achieved normal hearing that included the ability to hear whispers.

A. Eliot Shearer, M.D., Ph.D., an otolaryngologist at Boston Children’s Hospital and CHORD trial investigator, described the impact of the therapy firsthand: “In the pivotal trial, the one-time gene therapy demonstrated rapid, meaningful and consistent hearing responses, with most children achieving remarkable hearing improvements. I’ve witnessed firsthand my trial participant responding to their mother’s voice, dancing to music and interacting with the world, and these moments are now possible for more children born with this specific form of hearing loss.”

George Yancopoulos, Regeneron’s Chief Scientific Officer, framed the approval as a landmark moment for the field: “This unprecedented breakthrough in gene therapy has already proven to be life-changing for many of the children in our clinical trial and their families. We are honored to be the first company to ever offer such a gene therapy advance for free to those in the U.S., and it serves to highlight our belief that the biopharmaceutical industry can be a genuine force for good in the world.”

Janet DesGeorges, Executive Director of Hands and Voices, welcomed the new option while emphasising the importance of patient choice: “Families deserve access to balanced information and a range of options when navigating genetic hearing loss. As new treatments and innovations emerge, families can assess available options and choose the approach best suited to their unique circumstances.”

Otarmeni received Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA. Regulatory submissions are planned in additional markets. Patients seeking access information can contact Regeneron’s OnPath patient support program at 1-866-500-GENE.

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Related Topics and Keywords

hearing loss, otarmeni, regeneon