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 trial of 20 kids, 16 had improvements in hearing about five months after treatment. Five of 12 who were followed for at least 11 months had their hearing essentially restored to normal.“It’s miraculous,” said Kerri, the mother of a clinical trial participant who requested that her last name be withheld. 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.”AdvertisementAdvertisementThe 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 companies’ 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.Yancopoulos and Regeneron’s chief executive, Dr. Len Schleifer, appeared at the White House on Thursday as part of a Most Favored Nation drug pricing announcement; the Trump administration has struck dea …
