FDA Approves First Gene Therapy for Inherited Deafness

On April 23, the FDA approved Otarmeni, the first gene therapy for otoferlin deafness, a rare congenital condition affecting about 50 US babies annually. Developed by Regeneron, the treatment replaces a mutated gene causing faulty otoferlin protein, which prevents sound signals from reaching the brain. Delivered via surgery into the cochlea, the therapy improved hearing in 80% of 20 children (ages 10 months to 16 years) within a year; 42% could detect whispers. Regeneron plans to provide it free in the US. Critics from the Deaf community argue it threatens Deaf culture, with bioethicist Teresa Blankmeyer Burke of Gallaudet University warning it could reduce the Deaf population. Historian Jaipreet Virdi of the University of Victoria says it reinforces the idea that deafness must be fixed. A separate Chinese trial published in Nature on April 22 recovered hearing in 90% of 42 participants, including adults. John Germiller of Children's Hospital of Philadelphia suggests future treatments for progressive hearing loss. The therapy uses the same surgical approach as cochlear implants, which often produce robotic sound.