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Can gene therapy restore hearing in children with inherited deafness? What FDA approval means
FDA approves first gene therapy for inherited deafness, targeting OTOF mutations.
The US Food and Drug Administration has approved the first gene therapy designed to treat inherited deafness, marking a major step forward in treating rare genetic hearing conditions in children. According to CNN, the one-time treatment targets mutations in a gene known as OTOF, a rare condition that affects a small number of babies each year.
The therapy is designed to address the root genetic cause of hearing loss rather than relying on assistive devices.
The treatment has been developed by Regeneron Pharmaceuticals and is administered through a surgical procedure similar to cochlear implantation.
How effective is the treatment?
Early clinical trial results suggest the therapy can significantly improve hearing in children born without it. CNN reported that out of 20 children treated, 16 showed improvements in hearing within about five months.
Among those monitored for a longer period, some children experienced near-normal hearing levels, indicating the potential of the therapy to restore hearing function in certain cases.
Parents involved in the trial described the outcomes as life-changing.
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What makes this therapy different
Unlike traditional approaches such as hearing aids or cochlear implants, gene therapy works by delivering a functional copy of the faulty gene directly into the inner ear.
This approach aims to correct the underlying genetic issue, offering the possibility of long-term improvement after a single treatment. The therapy was also found to be safe in trials, with side effects mainly linked to the surgical procedure rather than the gene therapy itself.
Cost and accessibility
Regeneron said it plans to offer the therapy free of cost to patients in the United States. However, families may still need to cover costs associated with the surgical procedure used to deliver the treatment.
The company has also indicated plans to seek regulatory approvals in other countries, though it has not clarified whether the therapy will be free outside the US.
