FDA Approves First Gene Therapy for Inherited Deafness, Regeneron to Offer It Free in US
The FDA approved Otarmeni on April 23, the first gene therapy for genetic hearing loss. Developed by Regeneron, the treatment restored hearing in 80 percent of trial participants and will be offered at no cost to US patients.
The U.S. Food and Drug Administration approved Otarmeni on April 23, 2026, making it the first gene therapy ever cleared for the treatment of inherited deafness.
Otarmeni, developed by Regeneron Pharmaceuticals in collaboration with Decibel Therapeutics, targets severe-to-profound sensorineural hearing loss caused by biallelic variants in the OTOF gene. The condition affects approximately 50 babies in the United States each year and accounts for 2 to 8 percent of inherited, non-syndromic congenital hearing loss.
The therapy uses dual adeno-associated virus vectors to deliver a functional copy of the OTOF gene to inner hair cells in the cochlea. It is administered as a single surgical injection per ear.
In a clinical trial of 24 pediatric patients aged 10 months to 16 years, 80 percent of evaluable participants experienced improved hearing. Some achieved essentially normal hearing, including the ability to hear whispers, with benefits lasting at least two years.
Regeneron announced it will offer the therapy free of charge to patients in the United States. Co-founder and president Dr. George Yancopoulos said the company aims to "make an example of how science can really deliver a gift to people."
The FDA approved the therapy in 61 days under its Commissioner's National Priority Voucher pilot program, tying for the fastest Biologics License Application approval in modern FDA history.
Some scholars in the Deaf community have raised concerns that the therapy could reinforce the view of deafness as a problem requiring elimination rather than a form of human diversity.
