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Regeneron wins FDA approval for first gene therapy for genetic hearing loss
Clinical trial data showed 90% of participants improved, and some children later learned to speak, researchers said.
- Researchers reported Wednesday in the journal Nature that experimental gene therapy successfully restored hearing in people born with a rare form of deafness caused by OTOF gene mutations.
- The OTOF gene encodes otoferlin, a protein critical for translating sound vibrations into electrical signals the brain can interpret; without it, children are born completely deaf.
- About 90 percent of recipients saw hearing improvements, with the study involving participants aged nine months to 32 years and reporting no serious side effects or dose-related toxicity.
- Recipients showed improved speech perception, with some children learning to speak for the first time; gene therapy may eventually prove superior to mechanical Cochlear implants requiring maintenance.
- Regeneron Pharmaceuticals plans to file for regulatory approval, potentially becoming the first gene therapy for deafness to clear the Food and Drug Administration within the next year.
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First-ever gene therapy for hearing loss gets FDA approval: ‘Groundbreaking’
NEWYou can now listen to Fox News articles! 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 t…
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