US health authorities have approved Otarmeni, the first gene therapy designed to treat a rare form of genetically inherited deafness. Developed by the American biotech company Regeneron, this therapy targets mutations in the OTOF gene, which cause severe to profound deafness in approximately 50 newborns each year in the United States.
Administered as a single injection into the inner ear, the treatment is available to both children and adults. Regeneron announced that it will be provided free of charge to eligible US patients, a significant decision in a field where gene therapies often cost millions of dollars.
In a clinical trial involving 20 children aged 10 months to 16 years, more than 80% experienced a significant improvement in hearing. Families hailed the breakthrough as "incredible," noting the rapid progress in sound perception.
For Dr. Eliot Shearer of Boston Children’s Hospital, the FDA’s accelerated approval marks “a new era” in the treatment of genetic hearing loss, paving the way for the restoration of natural hearing in some patients.
 Pascal Lemontel
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24/04/2026 | 
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