The Food and Drug Administration (FDA) has approved Regeneron’s Otarmeni, the first gene therapy for genetic hearing loss. The drug targets a rare condition affecting about 50 babies born in the U.S. each year, caused by a mutation in the OTOF gene, which prevents the production of otoferlin—a protein essential for transmitting sound signals to the brain.
Otarmeni works by delivering a functional OTOF gene to the inner ear, restoring hearing in some patients. The approval was based on a clinical trial involving 20 children, where 16 showed improved hearing, with five developing the ability to detect whispers. Side effects included middle ear infections, nausea, vomiting, and dizziness.
The FDA granted the approval under its Commissioner’s National Priority Voucher program, which fast-tracks drug reviews for rare conditions. Regeneron announced it will offer the therapy for free to U.S. patients.
Dr. Eliot Shearer, a pediatric otolaryngologist at Boston Children’s Hospital, called the therapy ‘life-changing’, noting that it provides continuous hearing without the limitations of cochlear implants. Meanwhile, Zheng-Yi Chen, a researcher at Mass Eye and Ear, described the approval as a ‘landmark’ in hearing loss treatment.
Sierra Smith, whose son Travis was treated in an experimental trial, reported that he startled at sounds for the first time after receiving the therapy. The FDA’s decision was also highlighted by President Donald Trump during an Oval Office announcement about a drug pricing deal with Regeneron.