Otarmeni gene – A new gene therapy, Otarmeni, has received accelerated approval from the U.S. Food and Drug Administration to treat a rare genetic hearing loss. For Sierra Smith and her son Travis—born deaf—surgery at 18 months has meant startling, measurable progress and a r
When Sierra Smith pulled into the drive after weeks of waiting, she laughed loudly as she chatted with a friend—just to have her son, Travis, jolt in the back seat at a sound he hadn’t been able to hear before.
Travis. now 2 and a half. was born with hearing loss and had the Otarmeni gene therapy surgery when he was 18 months old. Otarmeni, a surgically-delivered drug developed by Regeneron, recently received accelerated approval from the U.S. Food and Drug Administration to treat a rare, genetic hearing loss condition.
For Smith, of East Greenbush, New York, the change wasn’t abstract. “He was such a mute baby, very serious,” she said. “Now he babbles. uses different pitches.” In the weeks after the procedure. hearing tests showed progress—but she described the early signals as something she could feel in ordinary moments.
Travis has begun socializing with other kids at library story hour and bopping along to music. Smith said he “loves Bruno Mars.” Language acquisition is still ahead. she added. but she hears the right trajectory: he’s making the sounds he needs and receives speech therapy three times a week. “I know it’s coming.”.
The approval matters because Otarmeni has moved from experimental promise toward a recognized treatment pathway for children who meet a specific genetic diagnosis. Researchers and Regeneron leadership say early results in clinical studies were fast and dramatic for those able to benefit.
“What we saw in clinical studies was that in a few weeks. children started to hear; within a few months. they could hear whispers. ” said Jonathon Whitton. head of the auditory global program for Regeneron. “These are kids that before the surgery, wouldn’t hear a gas-powered lawnmower if they were standing next to it.”.
That speed is part of why Smith called access “a miracle.” She said when she first learned Travis could benefit from the experimental treatment. she was desperate to figure out how to pay. “I was ready to sell anything. take out a million loans if my child could hear me say ‘I love you. ‘” Smith said. Then the surgeon explained it was a clinical trial, so it was free.
Travis’ surgery was performed by Dr. Lawrence Lustig. chair of the Department of Otolaryngology – Head and Neck Surgery at the Columbia University College of Physicians and Surgeons and otolaryngologist-in-chief at NewYork-Presbyterian Hospital/Columbia University Medical Center. Smith said that detail brought her father to tears—relief replacing the fear of whether the family could ever make the treatment happen.
Otarmeni is also drawing attention for what it costs in the United States. Regeneron made Otarmeni free in the U.S. under the FDA’s National Priority Voucher program, and it is the first gene therapy approved under that program. The pricing for the rest of the world has yet to be set.
“…We just thought it was the right thing to do here,” said Christos Kyratsous, Regeneron’s senior vice president, genetic medicines, internal and research. He said the decision was meant as “a nice demonstration about the power of science.”
Otarmeni targets a specific biology—and that specificity is shaping how families can find out whether their child might qualify.
In the U.S., about 1 in 500 children are born with hearing loss. The particular condition Otarmeni treats is far rarer: it’s linked to OTOF-related hearing loss caused by mutations in the OTOF gene. It’s believed to affect about 50 newborns per year in the U.S. In that disorder. the person lacks a functional otoferlin protein. which is critical for communication between the sensory cells of the inner ear and the auditory nerve.
Travis was born six weeks early. He failed the newborn hearing test in the NICU. and because he experienced several birth complications. his hearing loss was first categorized as auditory neuropathy. Smith pushed for genetic testing anyway. and that’s how his case was discovered to be a “perfect candidate” for Otarmeni.
For families trying to understand why genetic testing matters. Whitton described the bottleneck: “For many rare diseases. it’s hard to know how may cases are out there. because there aren’t many getting thorough genetic tests.” He added that with an approved gene therapy now available for a certain kind of hearing loss. more children could end up tested.
Conceptually. Whitton said. the treatment is straightforward: “Put the thing in that’s missing.” Otarmeni is an “in vivo” therapy. meaning the gene therapy is placed into the body’s cells. The gene therapy is surgically delivered in the inner ear. similar to where a cochlear implant is placed. but instead of attaching a device. the missing protein is introduced.
Whitton described how Regeneron created the “code” to make the protein needed for the condition. But getting that code into the right cells is the hard part. He said the company deciphered the code that alerts a cell to “turn on” protein-making capabilities. then built a “shell” to package the code and deliver it to the correct cell type in the inner ear.
To carry the instructions into those cells. the package is attached to a common adenovirus that acts as an “escort.” Whitton said most people already have antibodies to the virus. and he emphasized that it is “not a virus that gets you sick.” The protein shell is then injected to the right spot in the inner ear. When the shell reaches the right cell type, he said, it opens and delivers the instructions.
Kyratsous said not all gene therapies share the same delivery approach. “For many of these genetic medicines you have the payload you want to deliver into the cell, and then you choose the chauffer to get it into the cell.”
Regeneron’s executives framed Otarmeni as part of a broader push into genetic medicines. including programs aimed at common and difficult conditions. Whitton said the company has been focused on understanding the underlying genetics of human diseases “since the beginning of the company. ” and he tied that foundation to “transformational medicines.”.
The company is exploring gene therapy approaches for other areas. including a Parkinson’s disease program exploring how to get new medicines to the brain. and a treatment for fatty liver disease that would deliver genetic instructions via IV and route them through the liver. where a package of instructions would be dropped off.
But Whitton said there is a key difference in how these therapies work. While Otarmeni gives directions to create a missing protein, other treatments would turn down production of certain proteins. “The packages you deliver to the body, some create proteins, some silence proteins,” Whitton said.
Kyratsous described the research cycle in terms that don’t hide the setbacks. Genetic medicine. he said. takes years of experiments that may not directly lead to success but still provide building blocks for future wins. “It’s a road with a lot of turns and a lot of failures,” he said. “When we fail. we are trying to understand why we fail.” He said successes like Otarmeni are “easier learning. ” even as failures far outnumber the wins.
For Kyratsous, the human payoff is where the long work lands. “Seeing the direct connection of work to successful treatment in a person. ” he said. “definitely it’s a great feeling to get that once in a while.” He pointed to Smith’s son as the sort of outcome research can deliver—“Just look at little Travis.”.
Smith understands the risk that came with being among the first to try. “It was scary to allow her son to be a medical pioneer and let a doctor operate on his head. ” she said. while also saying she feels proud of Travis’ role in helping other children. “Every surgery has to be done for the first time,” she said. “He’s helping other kids and he doesn’t even know it yet.”.
The story of Otarmeni’s accelerated approval is now tied to everyday sounds—laughter in a car, a child’s startled reaction, and the steady return of hearing that Smith says she can see even as language itself takes more time to catch up.
Otarmeni Regeneron FDA accelerated approval gene therapy genetic hearing loss National Priority Voucher program Sierra Smith Travis OTOF-related hearing loss Columbia University NewYork-Presbyterian