FDA Approves Groundbreaking Gene Therapy for Rare Deafness

On April 23, 2026, the U.S. Food and Drug Administration (FDA) approved a gene therapy capable of curing a rare inherited form of deafness.

In a news release, the FDA stated that “Following the publication of powerful results of hearing restoration in the New England Journal of Medicine, the FDA acted swiftly to grant a national priority voucher for an accelerated review.”

“The maker of the therapy, Regeneron, plans to provide it free to any child who needs it,” New York Times’ Gina Kolata reported. “The therapy called Otarmeni, is intended for children with otoferlin deafness, a rare form of hearing loss caused by a mutation in a single gene. The mutation destroys a protein in the inner ear that is needed to transmit sound to the brain.”

“The decision, while only immediately affecting people born with a very rare form of genetic deafness, is being hailed as a milestone in the quest to treat hearing loss,” wrote Rob Stein for NPR. “The gene provides instructions to make the otoferlin protein, which is necessary for hair cells in the inner ear to transmit sound to the brain. Most of the patients began to hear for the first time within weeks, with the quality of their hearing improving over the following months, according to Regeneron.”

“Through the national priority voucher pilot program, the agency is accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions—such as novel dual vector gene therapies and combination products requiring coordination across multiple offices and centers—in significantly shortened timeframes,” said FDA Commissioner Marty Makary, M.D., M.P.H in a statement.