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 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.”

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 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 deals now with 17 of the largest pharmaceutical companies aiming to bring US drug prices more in line with the lower prices paid in peer nations in Europe and Asia.

Regeneron said it plans to file for regulatory approval of the gene therapy in countries other than the US but didn’t say whether it 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. The company noted that costs of receiving the therapy may include out-of-pocket costs related to the surgical procedure, which isn’t performed by Regeneron.

Reaching more people

Although Otarmeni treats mutations that make up about 1% to 3% of cases of genetic hearing loss at birth, doctors expect that the approval of the first gene therapy will spur more work and investment into the field, broadening treatment options in the future.

Already, multiple companies and academic groups have been working on gene therapies targeting OTOF, despite it affecting so few people, with many showing similarly strong results.

Because of that, the field has seen “an unbelievable interest in other forms of genetic deafness that are more common,” said Dr. Lawrence Lustig, chair of the Department of Otolaryngology-Head and Neck Surgery at the Columbia University College of Physicians and Surgeons, who treated several of the participants in the Regeneron clinical trial.

The venture capital investment “coming into biotech now to really help fund more common forms of deafness is really starting to come through,” Lustig added.

With OTOF mutations, children are born making a non-functional version of a protein called otoferlin. It’s necessary for transmitting sound messages from the tiny hairs in the inner ear to the brain. With a functioning copy of OTOF delivered with Otarmeni, they make the healthy otoferlin protein, and that connection is restored.

“It’s not like a little incremental change, which I think sometimes in science, things can feel like,” said Dr. A. Eliot Shearer, an otolaryngologist at Boston Children’s Hospital and assistant professor at Harvard Medical School who treated Miles and helped run Regeneron’s trial, in addition to one from Eli Lilly. “It’s completely life-changing.”

That was the case for Miles, who was born with profound hearing loss. His mom, Kerri, said that as he got older, he was a happy kid but had trouble interacting with his siblings and didn’t enjoy music or sitting to be read a story. They were thinking he could receive cochlear implants, electronic devices that stimulate the auditory nerve and enable people to hear certain sounds and understand speech.

But Kerri also pushed for genetic testing, and when the results showed a mutation in the OTOF gene, “that was really what changed everything,” she said.

“In the world of otolaryngology,” Kerri recalls their geneticist saying, “this could mean a lot for him.”

A Google search turned up news coverage of a little girl who’d received gene therapy and had miraculous results.

“And we were like, ‘oh, my goodness, this could be him. How is this real?’ ” Kerri said. “We had wrapped our heads around – ‘OK, we’re hoping for technology, we’re hoping for cochlear implants, and now we’re watching this little girl. … She can hear with her own ears.’ ”

Miles enrolled in the trial and had surgery to receive the gene therapy when he was 13 months old. About a month later, Kerri said, they noticed that he covered his ears when an ambulance went by. They realized he could hear the siren.

Now, “he loves to read books, especially dinosaur books,” Kerri said. “We were watching a movie the other night, and a song came on, and he had to jump down and start dancing because there was music, and music means I have to dance.”

It’s not clear how long the benefits of the gene therapy last; the hope is that they could be permanent.

A price tag of $0

A drug for such a rare disease, given only once in a patient’s lifetime, presents a pricing conundrum for pharmaceutical companies. Medicines can still cost hundreds of millions – or billions – of dollars to develop no matter how many people have the condition they address.

In the world of gene therapy, that’s meant price tags of as much as $2 million to $3 million for one person, which can often “price patients out of the market,” said Evan Seigerman, an analyst with financial firm BMO Capital Markets focused on the biopharmaceutical industry.

Regeneron had internal discussions about whether its hearing loss gene therapy needed a price tag like that as well, Yancopoulos said. Charging as much as $4 million for each child could result in revenue of $200 million to $400 million a year.

“That’s not chump change, and there were the people who were worried about nickels and dimes who were pushing us to do that,” Yancopoulos said. “But we believe that we are a very different type of company.”

Regeneron developed the gene therapy along with a smaller biotechnology company called Decibel Therapeutics, which it ultimately acquired in 2023 for $109 million. Steve Holtzman, the former CEO of Decibel, said the company recognized that this form of hearing loss “was not going to be a commercial market at all. But it gave us the place to start.”

When Regeneron bought Decibel, Holtzman recalls telling his successor that Regeneron was one of “very, very few companies in the world who will actually take this through” to human clinical trials, “because there is essentially no market for it.”

“They love the science,” Holtzman said.

And the experience of working on this gene therapy, Yancopoulos added, has helped provide a foundation for more to come, with potentially different pricing strategies. He noted that there are so many different rare diseases that they actually affect a large number of people, and “I’m not sure that it’s a great model that we should be charging millions of dollars for each rare disease treatment.”

“We’ll make our money at some point,” Yancopoulos said. “But we don’t have to make it from the very first example in these rare kids, where we can do so much good.”

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