FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition

By Meg Tirrell, CNN

(CNN) — The US Food and Drug Administration on Thursday approved the first gene therapy for inherited hearing loss, a one-time treatment that proved to be life-changing for a small number of children in a clinical trial who were born without hearing.

The condition it treats is rare, affecting up to about 50 babies born each year in the US with mutations in a gene known as OTOF. But the therapy’s effect can be profound: In a clinical trial of 20 kids, 16 had improvements in hearing about five months after treatment. Five of 12 that were followed for at least 11 months had their hearing essentially restored to normal.

“It’s miraculous,” Kerri, the mother of a clinical trial participant who requested that her last name be withheld, told CNN. Her 2-year-old son, Miles, had the gene therapy delivered to both ears in May. “You go from being told your child’s profoundly deaf and may only ever hear with technology to your child’s hearing right alongside his friends. … This is just amazing.”

The gene therapy is made by biotechnology company Regeneron, which also made news with its planned price for the medicine. Often, one-time gene therapies for rare conditions are priced by manufacturers at millions of dollars per patient in order to recoup their investment; Regeneron said Thursday that it will be free for patients in the US.

“We want to make an example of how science, and in this case biotech, can really deliver a gift to people – in this case, the gift of hearing,” Dr. George Yancopoulos, the co-founder and president of Regeneron, told CNN.

The medicine had been touted by FDA Commissioner Dr. Marty Makary as one the agency aimed to speed to market with a swift review under a new National Priority Voucher program. Regeneron also said it will attend an announcement at the White House on Thursday focused on a Most Favored Nation drug pricing agreement; the deals aim to bring US drug prices more in line with the lower prices paid in peer nations in Europe and Asia.

The company said it plans to file for regulatory approval in countries other than the US but didn’t say whether the therapy would also be made free to patients there.

The treatment, called Otarmeni, is administered using a surgical procedure similar to cochlear implantation, Regeneron said. It was shown to be safe in its clinical trial, with side effects related to those experienced from surgery. Regeneron noted that costs of receiving the therapy may include out-of-pocket costs related to the surgical procedure, which isn’t performed by Regeneron.

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