The FDA Approves the First-Ever Gene Therapy for Deafness, Which Aims to Restore Hearing in Kids With a Rare Inherited Condition
The FDA has approved the first-ever gene therapy for a rare form of congenital deafness, which improved hearing in 80% of pediatric participants in a clinical trial. The treatment, called Otarmeni, is designed to restore hearing in children with otoferlin deafness, a genetic condition affecting an estimated 50 babies born annually in the US.
The FDA approved the first gene therapy for a rare form of deafness on April 23. The treatment, Otarmeni, is for otoferlin deafness, caused by a mutated gene encoding a faulty protein. In a clinical trial, 20 children received the gene therapy, with 80% showing improved hearing within a year. The treatment involves delivering a working version of the gene into the cochlea, a snail-shaped organ in the inner ear. Regeneron, the treatment's maker, plans to provide Otarmeni for free to patients in the US. Otoferlin deafness affects about 2-8% of people with inherited deafness, with around 50 cases in the US each year.
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