The US FDA has approved the first gene therapy for inherited deafness, marking a significant milestone in treating rare genetic hearing conditions in children.
The one-time treatment targets mutations in the OTOF gene, a rare condition affecting a small number of babies each year.
Developed by Regeneron Pharmaceuticals, the therapy is administered through a surgical procedure similar to cochlear implantation.
Early clinical trial results show the therapy can significantly improve hearing in children, with 16 out of 20 treated children experiencing improvements within five months.
Some children even achieved near-normal hearing levels, indicating the potential of the therapy to restore hearing function in certain cases.
The therapy works by delivering a functional copy of the faulty gene directly into the inner ear, aiming to correct the underlying genetic issue.
Regeneron plans to offer the therapy free of cost to patients in the United States, although families may still need to cover costs associated with the surgical procedure.
