New York Company Behind First-Ever Free Gene Therapy for Hearing Loss

A newly approved gene therapy for a rare form of inherited hearing loss is being hailed as a historic breakthrough and it has strong ties to New York.

FDA Approves First-Ever Gene Therapy for Hearing Loss

The U.S. Food and Drug Administration on Thursday approved Otarmeni, developed by Regeneron Pharmaceuticals, marking the first gene therapy designed to restore hearing in patients born with a specific genetic mutation.

Regeneron, headquartered in Tarrytown, New York, said it will offer the treatment free of charge to patients in the United States, a rare move for a therapy that could otherwise cost millions.

How the Gene Therapy Works

The one-time treatment targets a mutation in the OTOF gene, which affects about 50 babies born in the U.S. each year. The gene is responsible for producing otoferlin, a protein that helps transmit sound signals from the inner ear to the brain. Without it, sound never reaches the brain, resulting in profound hearing loss.

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Until now, the primary option for children with this type of genetic deafness has been cochlear implants. While effective, those devices rely on external hardware and batteries and can’t fully replicate natural sound. Gene therapy, by contrast, works continuously once administered.

Clinical Trial Shows Promising Results

The FDA based its approval on a clinical trial involving 20 children. Sixteen showed improved hearing, and some were even able to detect very soft sounds, like whispers!

The therapy is delivered through a surgical procedure similar to cochlear implant surgery, where doctors access the inner ear and administer the treatment under general anesthesia. Side effects reported in the trial included ear infections, dizziness, nausea and vomiting.

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Experts say the approval could open the door to broader treatments for hearing loss in the future.