Lunsotogene Parvec Becomes First FDA-Approved Gene Therapy for OTOF-Related Hearing Loss

The FDA has granted accelerated approval to lunsotogene parvec-cwha (Otarmeni; Regeneron), making it the first and only gene therapy indicated for genetic hearing loss caused by variants in the otoferlin (OTOF) gene, according to a press release from Regeneron.¹ In tandem with the approval, the company announced that lunsotogene parvec will be provided at no cost to eligible individuals in the US, noting that out-of-pocket administration costs are not within the company’s control.

“[Lunsotogene parvec] is a huge scientific leap and is representative of Regeneron’s approaches to continually push the boundaries of science to benefit humanity,” George D. Yancopoulos, MD, PhD, board cochair, president, and chief scientific officer of Regeneron, said in a statement.¹ “This unprecedented breakthrough in gene therapy has already proven to be life-changing for many of the children in our clinical trial and their families. We are honored to be in the position to be the first company to ever offer such a gene therapy advance for free to those in the US and serves to highlight our belief that the biopharmaceutical industry can be a genuine force for good in the world.”

The therapy is indicated for pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 decibel hearing level [dB HL]) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the treated ear.¹

The accelerated approval was based on 24-week data from the pivotal, open-label, multicenter phase 1/2 CHORD trial (NCT05788536), which enrolled infants, children, and adolescents with OTOF-related hearing loss.² At 24 weeks, 80% of evaluable participants met or surpassed the primary end point—an average hearing threshold of 70 dB HL or less on behavioral pure-tone audiometry, a level that generally enables natural acoustic hearing and avoids the need for cochlear implantation.¹ With longer follow-up, 42% of evaluable participants achieved normal hearing, including the ability to hear whispers.

Formerly known as DB-OTO, lunsotogene parvec is an adeno-associated virus (AAV) vector–based gene therapy administered via a single intracochlear infusion. It is also the first gene therapy approved under the FDA Commissioner’s National Priority Voucher program, making it the second new molecular entity to receive that designation.

References

1. 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. News release. Regeneron Pharmaceuticals. April 23, 2026. Accessed April 23, 2026. https://investor.regeneron.com/news-releases/news-release-details/otarmenitm-lunsotogene-parvec-cwha-approved-fda-first-and-only

2. Valayannopoulos V, Bance M, Carvalho DS, et al. DB-OTO gene therapy for inherited deafness. N Engl J Med. 2026;394(11):1074-1083. doi:10.1056/NEJMoa2400521