FDA Approves First Gene Therapy for Deafness, Offers it Free

Travis Smith was born into complete silence, unable to hear his mother's voice or even recognize his own name.

His mother Sierra watched her baby grow up in a world without sound, struggling to connect with him. "My son was 100% deaf," she told reporters. "Didn't say mama, didn't even know he had a name."

But last June, everything changed when Travis received an experimental gene therapy called DB-OTO. Within months, the toddler's silent world transformed into one filled with music, laughter, and his mother's voice.

Today, the FDA approved that same therapy as Otarmeni, making it the first gene therapy ever approved for inherited hearing loss. And Regeneron Pharmaceuticals, the company behind it, announced they'll provide it completely free to all U.S. patients.

"He loves playing his little guitar. He loves listening to Bruno Mars," Sierra said of her now two-and-a-half-year-old son. "I'm so excited for him to say his first word."

Travis was born with a rare genetic condition affecting only 20 to 50 children each year. These babies lack a critical protein called otoferlin that acts like a bridge, transmitting sound from sensory cells to the brain. Without it, they're born into complete silence.

Regeneron's solution delivers a working copy of the gene directly into the ear during a surgery similar to cochlear implants. In clinical trials, nine out of 12 patients showed hearing improvement.

The therapy represents eight years of research and a complete expansion for Regeneron, previously known only for antibody treatments. The company acquired the technology through a $109 million purchase of Decibel Therapeutics in 2023.

The Ripple Effect

Otarmeni's approval marks a turning point in genetic medicine. By offering the therapy free, Regeneron is proving that pharmaceutical companies can prioritize patients over profits while still advancing groundbreaking science.

For families like Sierra's, the impact reaches far beyond hearing. She spent months advocating for genetic testing when doctors dismissed her concerns, citing Travis's premature birth and jaundice as more likely causes. Her persistence paid off, opening doors not just for Travis but for every child born with this condition going forward.

The company is already building teams to deliver the therapy nationwide starting day one, ensuring no family has to fight as hard as Sierra did to access this life-changing treatment.

Today, Travis is discovering a world of sound Sierra once feared he'd never experience. Tomorrow, dozens more children will have that same chance.