FDA Approves First Gene Therapy for Genetic Deafness, Offering Free Access in the U.S.
The U.S. Food and Drug Administration (FDA) has approved Regeneron Pharmaceuticals' Otarmeni (lunsotogene parvec-cwha), marking the first gene therapy for a form of genetic hearing loss. This therapy targets otoferlin-related hearing loss, a rare condition caused by mutations in the OTOF gene. The approval is based on the CHORD trial, where 80% of participants showed significant hearing improvement, with 42% achieving normal hearing. Regeneron has announced that the therapy will be available at no cost to U.S. patients, a move aimed at maximizing its reach and impact. The therapy involves a dual hybrid AAV system to deliver the OTOF gene, addressing a previously unmet medical need. 
