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A new therapy for genetic hearing loss has just been approved by the Food and Drug Administration (FDA).
The treatment, Otarmeni (lunsotogene parvec-cwha), is the first-ever dual adeno-associated virus (AAV) vector-based gene therapy — meaning it uses two harmless viruses to deliver the therapy into cells.
In an FDA news release, the agency called the therapy “groundbreaking,” noting its potential to treat both pediatric and adult patients with “severe to profound” hearing loss linked to the OTOF gene.
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Positive results from drug trials were published in The New England Journal of Medicine in 2025, confirming that the gene therapy “improved hearing in patients with OTOF-related deafness, enabling natural acoustic hearing and normalizing hearing sensitivity in three of 12 treated patients.”
A new therapy for genetic hearing loss has just been approved by the Food and Drug Administration. (iStock)
The FDA approval marks the first disease-modifying treatment for OTOF-related deafness.
This is the sixth drug approval under the FDA commissioner’s National Priority Voucher (CNPV) pilot program — and the first gene therapy to be approved through the initiative, according to the announcement.
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“Today’s approval is a significant milestone in the treatment of genetic hearing loss,” FDA Commissioner Marty Makary, MD, commented in a statement.
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“Through the national priority voucher pilot program, the agency is accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions – such as novel dual-vector gene therapies and combination products requiring coordination across multiple offices and centers – in significantly shortened timeframes.”
Food and Drug Administration Commissioner Marty Makary speaks during a press conference with Secretary of Health and Human Services Robert F. Kennedy Jr. at the Department of Health and Human Services in Washington, D.C., on Oct. 29, 2025, discussing administration plans to lower drug costs. (Annabelle Gordon/Reuters)
Otarmeni is administered surgically as a one-time treatment, using a needle and a tiny tube to deliver the medicine directly into the inner ear (the cochlea) in both ears.
The therapy works by delivering a healthy copy of the OTOF gene to key cells in the inner ear, helping to restore the protein otoferlin so that auditory signals can be sent to the brain, according to the FDA.
Side effects of Otarmeni may include middle ear infection, nausea, dizziness and procedural pain, according to the FDA. (iStock)
The treatment is intended for patients with preserved outer hair cell function and for those with no prior cochlear implant in the same ear. Side effects may include middle ear infection, nausea, dizziness and procedural pain.
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The FDA will host a public meeting on June 4 to discuss program implementation, eligibility criteria and other processes for distribution, according to the announcement.