A groundbreaking treatment that could enable deaf children to hear a whisper has been approved in the United States and will be offered to American children free of charge. The Food and Drug Administration (FDA) gave the green light to Regeneron's gene therapy, Otarmeni, on Thursday. This therapy targets a rare form of hearing loss that leaves babies completely deaf or only able to perceive very loud sounds from birth.
Understanding OTOF-Related Hearing Loss
The condition, known as OTOF-related hearing loss, affects approximately 50 babies in the US each year. It is caused by a mutation in the OTOF gene, which leads to the production of a malformed protein. This defective protein prevents sound signals from being transmitted to the brain, resulting in profound hearing loss.
Clinical Trial Results
In clinical trials involving 20 children with the mutation, 16 demonstrated significant improvements in hearing after receiving the therapy. Notably, five of these children were able to hear a whisper for the first time. Experts have hailed the treatment as life-changing for families, and Regeneron's vice president described it as the beginning of a new era in hearing loss treatments.
A Mother's Perspective
Sierra Smith, whose two-and-a-half-year-old son Travis was treated with the gene therapy, shared her family's transformative experience. She said: 'Watching him be able to interact with other children and even him knowing his name now and turning when I say his name is the craziest thing. I can tell him how much I love him — it's such a different world.'
Comparison with Cochlear Implants
Before this new treatment was approved, the only option for children with OTOF-related hearing loss was cochlear implants. While these implants restored the ability to hear speech and music, they did not allow for softer sounds. Unlike the gene therapy, cochlear implants do not provide 24/7 hearing and require batteries to function.
How the Therapy Works
The OTOF gene codes for otoferlin, a critical protein in the ear's inner hair cells essential for transmitting sound signals to the brain. In the condition, a mutated version of this protein is produced, leaving patients unable to hear anything or only very loud sounds. The new treatment uses a modified virus to deliver a working copy of the OTOF gene to the cells, restoring hearing. The virus is infused into the cochlea, the bony cavity within the inner ear, during a surgery similar to that required for cochlear implants, which can be given to children from nine months old.
Side Effects
In the clinical trial, side effects of the medication included infection or inflammation of the ear, vomiting, nausea, and dizziness.
Government and FDA Support
The approval was touted by President Donald Trump, who announced that the medication would be available via TrumpRx. This is part of a wider deal Regeneron signed with the administration to offer its medicines at lower prices for the Medicaid program and for cash payers. Trump stated: 'I'm thrilled to announce that one of the most respected pharmaceutical companies anywhere in the world, frankly, I know it very well is Regeneron, and it's agreed to offer their prescription medications at heavily discounted most favored nation prices.'
FDA Commissioner Dr. Marty Makary commented: 'Today's approval is a significant milestone in the treatment of genetic hearing loss. Through the national priority voucher pilot program, the agency is accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions in significantly shortened timeframes.'
The treatment was approved via the national priority voucher program, which accelerates review times for breakthrough treatments from 10 to 12 months down to one to two months. More than 50 million Americans suffer from hearing loss, estimates suggest, making it the third most common chronic condition in the country.
