The American biotech company Regeneron recently received approval from its health authorities to market its gene therapy medicine for people with deafness related to the OTOF gene.
The gene therapy developed in the United States aims to treat patients with a specific mutation of the OTOF gene that causes bilateral and generally profound deafness. It affects 20 to 50 new individuals each year in the United States.
The medicine, named Otarmeni by Regeneron, aims to replace the faulty gene with a functional copy. The American health agency, the Food and Drug Administration (FDA), accepted the gene therapy medicine into its “fast track” program in October 2025, which reduces the evaluation time to a few months from an average of one year for treatments that meet the public health priorities of the United States. And on April 23, the FDA granted approval for Otarmeni to be marketed through the standard process and not as an experimental treatment. The agency specified that the safety and efficacy were demonstrated in a clinical trial involving 24 children aged 10 months to 16 years. The 20 evaluable patients showed an improvement in their hearing.
The FDA’s authorization may be reviewed based on the patients’ progress. The medicine will be provided free of charge by Regeneron in the United States.
