American health authorities announced on Thursday, April 23, the approval of a unique gene therapy for a genetic form of deafness, a breakthrough that could pave the way for other treatments for these hearing deficits.
With two to three children per thousand born in the United States with hearing impairment and more than half of these early deafness cases believed to be genetic, the development of targeted gene therapies is generating high hopes.
Developed by the American biotechnology company Regeneron, this treatment named Otarmeni targets a rare type of deafness, affecting only about 50 new births per year in the United States. It can now be offered to children and adults suffering from severe to profound deafness linked to certain mutations in the OTOF gene, responsible for a protein that transmits auditory signals from the inner ear to the brain.
While gene therapies are usually extremely expensive, especially in the United States where they can cost millions of dollars per patient, Regeneron surprised many by announcing that it plans to offer this treatment for free to eligible American patients.
A revolutionary treatment
To be administered once in the form of an injection into the ear by a surgeon, this innovative therapeutic solution has been hailed as revolutionary by parents of affected children. “It’s just incredible,” said Sierra Smith, a young mother of a boy named Travis who benefited from the treatment.
“He didn’t know his own name. He couldn’t hear me tell him how much I love him. And now, thanks to Regeneron and this incredible operation, he can listen to music, and he loves it, he loves to dance,” she recounted from the White House during an event celebrating an agreement between the Trump administration and the pharmaceutical company on the prices of other medications.
A clinical trial conducted on 20 pediatric patients aged 10 months to 16 years showed significant improvement in hearing for at least 80% of them after a few months.
The approval of this innovative treatment by the American Food and Drug Administration (FDA) “marks a new era in the treatment of genetic forms of hearing loss, where it is now possible to restore continuous natural hearing,” praised Dr. Eliot Shearer from Boston Children’s Hospital, who participated in the clinical trial, in a statement.
