The U.S. Food and Drug Administration (FDA) has approved the first-ever gene therapy for inherited hearing loss.
The treatment, called Otarmeni, is approved to treat a type of hearing loss caused by mutations in the OTOF gene, which encodes a protein called otoferlin. Cells in the inner ear require otoferlin to convert vibrations into signals that the brain can interpret. When people have two defective copies of the OTOF gene (one from each parent), this line of communication between the inner ear and the brain is severed, resulting in severe to profound hearing loss.
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Although some participants improved to the point where they could hear whispers, all participants who responded to the therapy achieved a level of hearing that would not normally require a cochlear implant. This means using a device to bypass the inner ear and restore some hearing. Many people with this type of hearing loss receive cochlear implants, but the implants do not fully replicate natural hearing and require maintenance over time.
Based on trial results, Otalmeni has been approved for both children and adults with OTOF-related hearing loss, treatment maker Regeneron announced Thursday (April 23). The company said the treatment itself will be free for U.S. patients, but does not include out-of-pocket administrative fees that may be determined by the patient’s doctor or insurance.
“FDA approval of this gene therapy is a landmark moment for the field and, most importantly, for patients,” said Chen Yi Chen, associate scientist at Mass Eye & Year’s Eaton Peabody Institute and associate professor at Harvard Medical School. Chen is participating in a similar gene therapy trial for OTOF-related hearing loss in China.
Chen said the trial data “convincingly demonstrates both safety and efficacy.” He added that the approval process was very quick, taking less than three years from when it was administered to the first patient in 2023. (Otalmeni was approved through a special FDA “fast track” process, in part because there were no existing treatments for OTOF-related hearing loss that addressed the underlying cause.)
This speed “highlights both the robust clinical results and the urgent unmet medical need for children with OTOF-mediated congenital hearing loss,” Chen told Live Science. “We are very encouraged by this milestone, which will serve as a catalyst to accelerate the development of future gene therapies for hearing loss.”
OTOF-related hearing loss affects approximately 50 newborns per year in the United States. This new treatment is approved for people who are missing two copies of the OTOF gene and have no history of therapeutic cochlear implants in their ears. Because installing these implants causes damage to the inner ear, gene therapy is unlikely to be effective. However, people with only one cochlear implant can receive gene therapy in the opposite ear.
Patients seeking treatment must also have their outer hair cells intact. Outer hair cells are special cells in the ear that act as amplifiers that increase the movement of the eardrum in response to sound.
An international trial testing Otarmeni is currently underway, recruiting children under 18 in the United States, United Kingdom, Spain, Germany and Japan.
“I witnessed first-hand as trial participants responded to their mothers’ voices, danced to music, and interacted with the world. Moments like this are now possible for more children born with this particular form of hearing loss,” Dr. A. Elliot Shearer, an otolaryngologist at Boston Children’s Hospital, an associate professor at Harvard Medical School, and a trial participant, said in a Regeneron statement.
This article is for informational purposes only and does not provide medical advice.
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