A New Era in Hearing Care: FDA Approves OTOF Gene Therapy

In the United States, mutations in the OTOF-gene (otoferlin gene) account for between one percent and eight percent of non-syndromic congenital hearing loss (Ford et al., 2023). These losses are often profound. OTOF mutations are also the leading cause of auditory neuropathy spectrum disorder (Ford et al., 2023). Otarmeni, created by Regeneron Pharmaceuticals, is the first and only FDA-approved gene therapy for hearing loss. This drug uses an inactivated virus to deliver working OTOF genes directly to the cochlea. In clinical trials, 80 percent of participants had improved thresholds from profound to better than 70 dB HL; 70 percent demonstrated a present auditory brainstem response at 90 dB nHL; and 42 percent achieved hearing thresholds within normal limits (Regeneron, 2026). This medication is delivered under general anesthesia via intracochlear infusion. According to the drug manufacturer, the medication will be free to all patients and is currently available.

References

Ford, C. L., Riggs, W. J., Quigley, T., Keifer, O. P. Jr, Whitton, J. P., Valayannopoulos, V. (2023). The natural history, clinical outcomes, and genotype-phenotype relationship of otoferlin-related hearing loss: a systematic, quantitative literature review. Human Genetics, 1429-1449.

Regeneron (2026). Otarmeni™ (lunsotogene parvec-cwha) approved by FDA as first and only gene therapy for genetic hearing loss; Regeneron to provide Otarmeni for Free in the U.S. (press release).