The Food and Drug Administration (FDA) approved the first gene therapy for genetic hearing loss to enter the market in the US. The FDA cleared Regeneron’s Otarmeni for children born with hearing loss caused by gene mutations, the company said Thursday, according to Reuters. Regeneron plans to make the therapy available within weeks and to offer it at no cost to patients in the US, according to NPR.
The decision follows clinical results in patients born with a specific defective gene. The healthy gene provides instructions for making a protein necessary for inner-ear hair cells to transmit sound signals to the brain.
"It's the first time in history there's a new drug for hearing loss," said Boston-based researcher Zheng-Yi Chen, according to NPR. His team reported promising results with a similar approach. "I think it's an historical event, a landmark, a great development for the whole field," he said.
Viruses delivering a healthy gene version
In the treatment, doctors infuse billions of viruses into the ear after making a small incision behind the ear to open a small hole in the skull. The viruses delivered a healthy version of the gene that had been split in half to fit inside the virus. Most patients began to hear within weeks, with 80% achieving at least significant hearing restoration and 42% reaching normal hearing, including the ability to hear whispers. Improvements have been sustained for at least two years so far.
Boy's first reaction to sound
Travis Smith was born profoundly deaf. After doctors delivered the gene therapy to his inner ear his mother, Sierra Smith, noticed him startle at loud sounds and begin to wake to voices and noises. These were his first responses to sound.
Sierra and her son attended the announcement at the White House Thursday. US President Donald Trump Trump noted Travis can now hear his mother say “I love you.”
"He didn't know his name. He couldn't hear me tell him how much I love him. And now with Regeneron and this amazing surgery, he can listen to music. And he loves it. And he loves to dance," Sierra said.