DNA Therapy Turns the Sound Back On

Regeneron's Otarmeni wins FDA approval as the first dual-virus gene therapy to restore normal hearing levels in children with a rare mutation

26 Jun 2026

Approval from the US Food and Drug Administration has cleared the first dual adeno-associated virus gene therapy to treat a rare form of hereditary deafness. Developed by Regeneron, the treatment, called Otarmeni, targets hearing loss caused by mutations in the OTOF gene. It is the first authorized gene therapy to restore a neurosensory function to normal levels.

Data from the CHORD Phase 1/2 clinical trial drove the regulatory decision after evaluating 20 pediatric and adolescent patients. Eighty percent of participants met the primary endpoint by registering hearing levels of 70 decibels or better. After extended monitoring, 42 percent of the cohort achieved fully normal hearing.

"This unprecedented breakthrough in gene therapy has already proven to be life-changing for many of the children in our clinical trial and their families," said George D. Yancopoulos, board co-chair, president and chief scientific officer of Regeneron.

The biotechnology company announced it will provide the therapy free of charge to eligible patients in the US. This commercial strategy addresses widespread concerns over the affordability of advanced genetic medicines. The decision could influence pricing frameworks across the wider market for rare disease treatments.

For the broader healthcare sector, the milestone introduces new competition for traditional interventions such as cochlear implants. The introduction of a single-intervention therapy capable of restoring durable sensory function will require healthcare systems and investors to adapt. Regeneron must now establish the clinical infrastructure necessary to identify patients and deliver the treatment.