04/24/2026
On April 23, the FDA announced approval for Otarmeni, a form of gene therapy for children born with genetic hearing loss. Regeneron, the company behind the breakthrough, has also committed to providing the therapy for free in the U.S. initially to ensure immediate access for eligible children. The impact of this ground-breaking announcement has sent waves across the hearing loss community.
"The recent approval of Otarmeni will be life-changing for children born with congenital hearing loss. By moving beyond assistive devices to address the genetic root of hearing loss, Regeneron is providing a definitive breakthrough for children born with otoferlin mutations," said Emilio Alonso-Mendoza, CEO of AG Bell. "This milestone reinforces our commitment to a world where every child has the opportunity to hear, speak, and thrive. This isn't just a new treatment; it is the dawn of a new era for children born with hearing loss."
New clinical results from therapy trials show that children born profoundly deaf began responding to sound within weeks of treatment. Unlike cochlear implants, which bypass the ear’s natural mechanics, this gene therapy delivers a functional gene directly into the cochlea using a modified virus—restoring the body’s ability to process sound naturally rather than amplifying it. It is important to note that the therapy is only for children with genetic hearing loss, specifically otoferlin mutations. Genetic testing is required to ensure the child is eligible for the gene therapy.
This represents a game-changing option for parents. It offers the potential for children to develop speech and language milestones alongside their hearing peers without the permanent surgical hardware of an implant. AG Bell applauds Regeneron's decision to offer the therapy for free to ensure all children with hearing loss get immediate access to the hearing care they need.
🔗 For more information on gene therapy, visit the AG Bell resource page: https://agbell.org/resources/gene-therapy/
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For every child who is deaf or hard of hearing (DHH), there is one or more reasons for their hearing loss. According to the CDC and other sources*, genetic factors are responsible for around 50-60% of childhood hearing loss.
