A breakthrough treatment that could allow deaf children to hear a whisper has been approved in the US — and is set to be offered to American children for free.
The Food and Drug Administration gave the green light to Regeneron’s gene therapy Otarmeni on Thursday, a treatment for a rare form of hearing loss that leaves babies completely deaf or only able to hear very loud sounds from birth.
The condition, known as OTOF-related hearing loss, affects about 50 babies in the US every year and is caused by a mutation in the OTOF gene, which causes a malformed protein to be made that stops signals from sounds being transferred to the brain.
In clinical trials involving 20 children with the mutation, 16 showed significant improvements in hearing after receiving the therapy.
Five of the children were also able to hear a whisper for the first time.
Experts have hailed the treatment as ‘life-changing’ for families, while Regeneron’s vice president said it marked the beginning of a ‘new era’ of hearing loss treatments.
Sierra Smith, whose two-and-a-half-year-old son Travis was treated with the gene therapy, said it had opened up a ‘different world’ to her family.
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.
OTOF-related hearing loss is a genetic condition where sounds are not transmitted from the ear to the brain (stock image)
‘I can tell him how much I love him — it’s such a different world.’
Before the new treatment was approved, the only option for children with the condition was cochlear implants, which restored the ability to hear speech and music but not softer sounds.
Unlike the gene therapy, the implants did not give children 24/7 hearing and required batteries to function.
The OTOF gene codes for Otoferlin, a critical protein in the ear’s inner hair cells that is essential for transmitting sound signals to the brain.
In the condition, a mutated version of this protein is made that leaves patients unable to hear anything or only very loud sounds.
In the new treatment, physicians use a modified virus to deliver a working copy of the OTOF gene to cells, which helps to restore hearing.
The virus is infused into the cochlea, the bony cavity within the inner ear, during a surgery similar to that needed for placing cochlear implants. These implants can be given to children from nine months old.
In the clinical trial, side effects of the medication included infection or inflammation of the ear, vomiting, nausea and dizziness.
The approval was yesterday touted by President Donald Trump, who said that the medication would be available via TrumpRx.
It was 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 said: ‘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 said: ‘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, a program to accelerate review times for breakthrough treatments from 10 to 12 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.
