FDA grants accelerated approval to Regeneron's Otarmeni, the first gene therapy for inherited deafness

Regeneron Pharmaceuticals/Otarmeni (DB-OTO)

The FDA granted accelerated approval to Otarmeni (lunsotogene parvec-cwha), a one-time gene therapy for severe-to-profound sensorineural hearing loss caused by biallelic OTOF mutations. In the pivotal CHORD trial, 80% of participants showed improved hearing, and 42% achieved normal hearing levels. Regeneron will provide the therapy at no cost to eligible U.S. patients.