FDA Grants Accelerated Approval for OTOF Gene Therapy
A one-time intracochlear gene therapy improved hearing outcomes in patients with OTOF-related sensorineural hearing loss in the phase 1/2 CHORD trial.
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By
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Kathryn Wighton
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April 23, 2026
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The FDA has granted accelerated approval for lunsotogene parvec-cwha to treat severe-to-profound sensorineural hearing loss due to OTOF gene variants.
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The therapy uses an adeno-associated virus vector to deliver a functional OTOF gene copy to cochlear hair cells, aiming to restore hearing.
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In the Phase 1/2 CHORD trial, 16 of 20 patients improved to 70 decibels or better at 24 weeks, indicating significant hearing restoration.
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The therapy is not recommended for patients with prior cochlear implants or those without accessible inner ear access.
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Adverse events included otitis media and dizziness, with surgical risks such as meningitis and facial nerve complications.
