FDA cleared Regeneron’s Otarmeni today, the first approved gene therapy for otoferlin-driven hearing loss. Regeneron’s giving it away for free.
Regeneron acquired the therapy through its $109 million buyout of Decibel Therapeutics in 2023. Otarmeni uses a viral vector to deliver a functional copy of the otoferlin gene into the inner ear. Otoferlin sits at the base of sensory hair cells and helps transmit sound waves to the brain. Children born missing both copies of the gene are profoundly deaf.
In the phase 1/2 CHORD trial, nine of 12 patients hit the primary endpoint for hearing improvement. The condition affects only 20 to 50 newborns per year in the U.S.
Otarmeni is also the first therapy cleared under the controversial Commissioner’s National Priority Voucher program, a designation aimed at accelerating rare disease treatments. That’s the part worth watching: the program is designed to pull forward rare disease medicines, and Regeneron’s approval here could serve as a template for other developers targeting ultra-rare indications.
This is Regeneron’s first gene therapy approval after eight years of building its genetic medicine division. Its pipeline now includes gene editing and RNA-based gene silencing tools. Regeneron is also investing in age-related and noise-induced hearing loss, conditions affecting millions.
Regeneron’s decision to give Otarmeni away is the first time any U.S. company has offered an FDA-approved gene therapy for free.
The commercial team is ready. Day one.
Sarah Chen