FDA Approves First Gene Therapy for Genetic Hearing Loss

Think of your ears like microphones. They pick up sound waves, convert them into electrical signals, and send them as messages to your brain. People with OTOF hearing loss, however, are born without a protein that’s essential to this process. When the otoferlin protein is missing or defective, these signals fail to transmit across the synapse to the auditory nerve. Sound enters their ears normally, but cannot reach their brains.

This type of hearing loss is caused by changes or mutations to the OTOF gene. It triggers severe to profound hearing loss in those born with this genetic mutation. A child must inherit a nonworking copy of the gene from both parents to show symptoms. OTOF accounts for 2 to 8% of all congenital, inherited, non-syndromic hearing loss cases worldwide, about 200,000 children and adults.

“For decades, our treatments for OTOF auditory impairment have focused on helping people hear better with hearing aids or cochlear implants,” says Adrien Eshraghi, M.D., MSc, FACS, co-director of UHealth’s Ear Institute, part of the University of Miami Health System.

Hearing aids amplify sound. Cochlear implant processors capture sound and transmit it to a receiver surgically implanted under the skin behind the ear. Signals are then sent to the auditory nerve of the brain, which interprets them as sound.

In April 2026, the U.S. Food and Drug Administration approved a new treatment option for patients with OTOF hearing loss.

“This is a historic moment in hearing care.”
– Dr. Adrien Eshraghi, co-director of UHealth’s Ear Institute

Otarmeni (lunsotogene parvec-cwha) is the first FDA-approved gene therapy for OTOF-related hearing loss. It is the only treatment to deliver a healthy copy of the OTOF gene directly into the inner ear (cochlea). This therapy is administered as a one-time surgical procedure similar to a cochlear implant surgery that prompts the production of functional otoferlin protein.

“Once the hearing cells receive the new gene, they can begin making the missing protein again. This allows the cells to send sound signals to the brain more normally, improving hearing in many patients,” Dr. Eshraghi says. “Instead of simply making sounds louder like a hearing aid, gene therapy aims to fix the underlying problem causing the hearing loss in carefully selected patients.”

Who may benefit from OTOF gene therapy?

In accordance with the FDA approval, Otarmeni is currently approved for use only in children and adults with:

• severe to profound inherited sensorineural hearing loss
• confirmed variants in both copies of the OTOF gene
• preserved outer hair cell function
• no prior cochlear implant in the same ear

Not everyone with hearing loss has this specific condition. In fact, more than 150 different genes can cause inherited hearing loss, and each may require a different treatment.

“This new gene therapy requires that the patient has healthy hearing cells present in the inner ear. If those cells are no longer functioning or have been lost, there may be nothing for the new gene to repair,” Dr. Eshraghi says.

So far, the best results of this new treatment are in young children. “A child’s brain is still developing and is especially good at learning to understand speech once hearing is restored. The earlier hearing is improved, the greater the opportunity for normal speech and language development,” he says. “Adults may also benefit, particularly if their hearing nerve and brain have continued to receive some sound information over the years. Because this treatment is so new, researchers are continuing to study how well it works at different ages and how long the benefits last.”

Where Patients Can Receive Gene Therapy

Otarmeni gene therapy is currently available exclusively at authorized treatment centers.

“UHealth has been a leader in cochlear implants, hearing restoration, hearing research, and clinical trials for many years,” Dr. Eshraghi says, who serves patients across Miami-Dade, Broward, and Palm Beach counties at the UHealth SoLé Mia Medical Center as well as locations in Miami and Plantation.

UHealth’s Ear Institute has helped restore hearing to more than 1,000 deaf children and adults. “As gene therapy becomes part of routine patient care, we are carefully evaluating opportunities to offer these groundbreaking treatments. Instead of simply helping people hear better, we hope to restore hearing by repairing the cells that are responsible for hearing.

“I believe this is only the beginning. Over the next 10 years, advances in gene therapy, gene editing, stem cell therapy, and regenerative medicine could completely change how we treat hearing loss,” he says. “Our goal is always to provide patients with access to the most advanced, safe and effective treatments available.”

To schedule an appointment with an expert at the UHealth Ear Institute, call 305-243-3564.

Written by Dana Kantrowitz. Reviewed by Adrien Eshraghi, M.D., MSc, FACS.

Tags: Dr. Adrien Eshraghi, FDA-approved gene therapy, hearing restoration, inherited hearing loss, otolaryngology in Miami