The Food and Drug Administration on Thursday approved a gene therapy that can cure a rare, inherited form of deafness. The treatment is the first to restore normal hearing in children who were born deaf. The maker of the therapy, Regeneron, plans to provide it free to any child who needs it.
“We wanted to make a statement,” Dr. George Yancopoulos, Regeneron’s chief scientific officer said on Thursday morning. He explained that the company wants to be sure its treatment “would be able to reach its full potential and help as many people as possible.
” Some gene therapies for other diseases, priced in the millions of dollars, have had dismal sales. The therapy called Otarmeni, is intended for children with otoferlin deafness, a rare form of hearing loss caused by a mutation in a single gene.
The mutation destroys a protein in the inner ear that is needed to transmit sound to the brain. Although otoferlin deafness accounts for just 2 percent to 8 percent of congenital hearing loss, the new treatment “is groundbreaking,” Dr.
Dylan Chan, a pediatric otolaryngologist at the University of California, San Francisco, said. He added, “This is the first time in history that there has been a medical therapy that has enabled deaf children to hear. ” We are having trouble retrieving the article content.
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