US Approves Innovative Gene Therapy for Hereditary Deafness

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US Approves Innovative Gene Therapy for Hereditary Deafness

US health authorities have approved the sale of a first-of-its-kind gene therapy for a type of hereditary deafness, a breakthrough that paves the way for other treatments targeting this hearing problem. The treatment is administered via a single injection into the ear by a surgeon.

The development of targeted gene therapies is generating considerable hope. In the United States, two to three out of every 1,000 children are born with hearing loss, and it is estimated that half of all cases of early-onset deafness are hereditary.

The new treatment, Otarmini, developed by the US biotechnology company Regeneron, targets a rare form of deafness that affects approximately 50 newborns annually in the United States.

The treatment will initially be available to children and adults with severe deafness linked to specific gene mutations in the OTOF gene, which is responsible for a protein that transmits auditory signals from the inner ear to the brain.

Although gene therapies are very expensive, especially in the United States where they can cost millions of dollars per patient, Regeneron has announced its intention to offer this treatment free of charge to eligible American patients. The treatment is administered via a single injection in the ear by a surgeon.