The FDA has approved Otarmeni, the first gene therapy for genetic hearing loss, through its National Priority Voucher program.
The therapy is indicated for pediatric and adult patients with severe-to-profound hearing loss tied to OTOF gene mutations, according to an April 23 agency news release. It is the first gene therapy approved under the program and was cleared 61 days after biologics license application filing, marking one of the fastest approvals in FDA history.
Otarmeni is a one-time treatment that delivers a functional copy of the OTOF gene to restore auditory signaling. In a clinical trial of 24 pediatric patients, 80% of evaluable participants experienced improved hearing.
The approval builds on earlier research showing strong efficacy for gene therapies targeting OTOF-related deafness. A prior study found about 90% of patients experienced hearing improvement following treatment.
The therapy addresses a condition with no prior disease-modifying treatments. OTOF gene variants account for 2% to 8% of inherited nonsyndromic hearing loss cases, the release said.
The FDA granted the therapy orphan drug, rare pediatric disease, fast track and regenerative medicine advanced therapy designations, and said continued approval may depend on long-term outcomes.
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