The Food and Drug Administration (FDA) has approved Regeneron’s Otarmeni, the first gene therapy for genetic hearing loss, which is significant for affected families. This groundbreaking therapy is designed to treat a rare form of hearing loss caused by a mutation in the OTOF gene, impacting about 50 newborns in the U.S. each year. The FDA granted approval under its Commissioner’s National Priority Voucher program, which accelerates the review process for certain therapies.
Dr. Eliot Shearer, a pediatric otolaryngologist, described the approval as life-changing for families with hearing-impaired children. Currently, the only other treatment available for genetic deafness is cochlear implants. These devices can restore sound but do not capture the full range of sound quality. In contrast, the gene therapy offers continuous hearing that is not dependent on batteries.
Otarmeni works by correcting the faulty OTOF gene, which is crucial for making a protein called otoferlin. This protein is essential for transmitting sound signals from the ear to the brain. The FDA’s approval was based on a trial involving 20 children with the OTOF mutation, where 16 showed improved hearing after a single dose.
While Regeneron will provide the drug at no cost to U.S. patients, the company does not control the administration costs, which are similar to cochlear implant surgery. The drug is currently not approved outside the U.S., and there is no price set for those living abroad. Sarah Emond, CEO of the Institute for Clinical and Economic Review, praised Regeneron for its strategy to ensure affordable access to this innovative therapy.
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