FDA Grants Accelerated Approval for OTOF Gene Therapy - Takeaways - MDSpire

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.

  • By

  • Kathryn Wighton

  • April 23, 2026

  • 3 min

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  • 1

    The FDA has granted accelerated approval for lunsotogene parvec-cwha to treat severe-to-profound sensorineural hearing loss due to OTOF gene variants.

  • 2

    The therapy uses an adeno-associated virus vector to deliver a functional OTOF gene copy to cochlear hair cells, aiming to restore hearing.

  • 3

    In the Phase 1/2 CHORD trial, 16 of 20 patients improved to 70 decibels or better at 24 weeks, indicating significant hearing restoration.

  • 4

    The therapy is not recommended for patients with prior cochlear implants or those without accessible inner ear access.

  • 5

    Adverse events included otitis media and dizziness, with surgical risks such as meningitis and facial nerve complications.

Original Source(s)

FDA Grants Accelerated Approval for OTOF Gene Therapy

  • by Kathryn Wighton

  • April 23, 2026

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