FDA Approves Otarmeni, the First Gene Therapy to Restore Hearing

The U.S. Food and Drug Administration (FDA) has approved Otarmeni (lunsotogene parvec-cwha), marking a major milestone in the treatment of certain types of genetic hearing loss. Developed by Regeneron Pharmaceuticals, this therapy is the first gene therapy approved in the U.S. that can restore a neurosensory function — hearing — to near-normal levels.

“The FDA approval of Otarmeni signals a new era in the treatment of genetic forms of hearing loss, where reinstating 24/7 natural hearing is now possible,” said CHORD trial investigator A. Eliot Shearer, M.D., Ph.D., otolaryngologist in the Department of Otolaryngology and Communication Enhancement at Boston Children’s Hospital, and associate professor of Otolaryngology-Head and Neck Surgery at Harvard Medical School.

Otarmeni is designed for patients with severe-to-profound hearing loss caused by mutations in the OTOF gene. This rare condition affects about 50 newborns in the U.S. each year. In these individuals, the inner ear structures are intact, but a missing or nonfunctioning protein prevents sound signals from reaching the brain.

Unlike traditional treatments such as hearing aids or cochlear implants, which amplify sound but don’t fully restore hearing, Otarmeni works by delivering a healthy copy of the OTOF gene directly into the inner ear. This allows the ear to process sound more naturally.

The approval is based on results from the CHORD clinical trial. Among 20 participants, 80% showed meaningful improvements in hearing within 24 weeks, reaching levels that may allow for natural hearing without cochlear implants. Even more notably, with longer follow-up, 42% achieved hearing levels considered normal — including the ability to hear whispers.

“In the pivotal trial, the one-time gene therapy demonstrated rapid, meaningful and consistent hearing responses, with most children achieving remarkable hearing improvements,” Shearer explained. “I’ve witnessed firsthand my trial participant responding to their mother’s voice, dancing to music and interacting with the world, and these moments are now possible for more children born with this specific form of hearing loss.”

The treatment is given as a one-time surgical procedure, similar to cochlear implant surgery. While side effects such as ear infections, dizziness and nausea were reported, the therapy showed promising safety results overall.

Otarmeni was granted accelerated approval, meaning additional research is ongoing to confirm long-term benefits. Importantly, the therapy will be made available at no cost to eligible patients in the U.S., though procedure-related costs may vary.

This approval represents a significant step forward, offering new hope for patients and families affected by genetic hearing loss.

“Connection and communication are at the heart of how we experience the world — whether that happens through listening and spoken language, sign language, the use of technology or a combination of approaches,” said Janet DesGeorges, executive director of Hands & Voices. “Families deserve access to balanced information and a range of options when navigating genetic hearing loss. As new treatments and innovations emerge, families can assess available options and choose the approach best suited to their unique circumstances.”