FDA Approves First Gene Therapy for Genetic Hearing Loss

The Food and Drug Administration approved Regeneron Pharmaceuticals' Otarmeni on Thursday, making it the first gene therapy approved to treat genetic hearing loss.

The treatment targets a very rare form of inherited deafness. Gene therapy works by replacing faulty genes that cause disease with healthy copies.

The FDA fast-tracked the approval under its new priority voucher program. The agency approved Otarmeni just 61 days after Regeneron filed its application, tying the record for fastest approval in modern FDA history.

This marks the sixth approval under the Commissioner's National Priority Voucher pilot program and the first gene therapy product approved through this pathway.

While the treatment addresses a rare condition, medical experts say it represents a major milestone. Success with genetic hearing loss could pave the way for gene therapies targeting other inherited conditions.