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The first gene therapy for OTOF-related genetic hearing loss won accelerated FDA approval

The peer-reviewed CHORD report found that nine of 12 children met the week-24 auditory endpoint, six could hear soft speech without assistive devices and three reached average normal hearing sensitivity. The FDA later reviewed an ongoing single-arm study with 24 treated children, 20 of whom were efficacy-evaluable, and granted accelerated approval to Otarmeni after 80% of evaluable participants improved.

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Stable ID
science-2026-otarmeni-otof-hearing-gene-therapy
Revision
science-2026-otarmeni-otof-hearing-gene-therapy.v1
Field
Medicine · Gene therapy and hearing
Evidence
Tier 1 · Peer reviewed: Yes
Record state
Current · FDA accelerated approval
Last checked

AI role

No substantive AI role was reported.

Record details

Problem or result
People with biallelic OTOF variants can have severe-to-profound congenital hearing loss because inner hair cells cannot transmit auditory signals normally.
Authors
Vassili Valayannopoulos et al.; CHORD Study Group
Institutions
Multicentre CHORD trial sites in North America and Europe; Regeneron Pharmaceuticals
Result date
April 23, 2026

Why it matters

Otarmeni is the first approved disease-modifying treatment for OTOF-related deafness and the first FDA-approved dual-AAV gene therapy.

Limits

Accelerated approval rests on a single-arm rare-disease trial and a hearing-sensitivity surrogate; continued approval may depend on durability and confirmation of speech-development and quality-of-life benefits. The indication is limited to molecularly confirmed biallelic OTOF variants with preserved outer hair-cell function and no prior implant in the treated ear.

Sources

  1. Primary: NEJM CHORD primary paper
  2. Primary: FDA approval announcement
  3. Primary: FDA product and review documents
  4. Independent: International expert consensus on hereditary-hearing-loss gene therapy

Correction and revision history

  1. 2025-10-12 — CHORD results published online in the New England Journal of Medicine.
  2. 2026-04-23 — FDA granted accelerated approval to Otarmeni, formerly DB-OTO.

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