The U.S. Food and Drug Administration has approved the first gene therapy to treat inherited deafness, marking a major step toward treating a rare genetic hearing disorder in children. The one-time treatment targets genetic mutations, CNN reports Gene A rare disease called OTOF affects a small number of babies each year.
The therapy is designed to address the underlying genetic causes of hearing loss rather than relying on assistive devices.
This treatment is made by Regeneron Pharmaceuticals and through a surgical procedure similar to a cochlear implant.
How effective is the treatment?
Early clinical trial results suggest the treatment can significantly improve Children’s hearing Born without it. CNN reported that 16 of the 20 children who received the treatment showed hearing improvement within about five months.
Among children monitored over time, some achieved near-normal hearing levels, suggesting the therapy has the potential to restore hearing function in some cases.
Parents who took part in the trial said the results had changed their lives.
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How is this therapy different?
Unlike traditional methods such as hearing aids or cochlear implants, gene therapy works by delivering a functional copy of the defective gene directly to the inner ear.
This approach is designed to correct the underlying genetic problem, offering the possibility of long-term improvement after a single treatment. The trial also found the therapy to be safe, with side effects mainly related to the surgical procedure rather than to the gene therapy itself.
Cost and accessibility
Regeneron said it plans to make the treatment free to U.S. patients. However, families may still have to pay the costs associated with the surgical procedures used to provide treatment.
The company also said it plans to seek regulatory approval in other countries, but has not clarified whether the treatment will be free outside the United States.
