FDA Approves First and Only Gene Therapy for Genetic Hearing Loss

Otarmeni™ (lunsotogene parvec-cwha), formerly known as DB-OTO, is the first and only in vivo gene therapy for OTOF-related hearing loss and will be made available by Regeneron for free in the U.S. UW faculty members Jay T. Rubinstein, MD, PhD, and James O. Phillips, PhD, were investigators on an associated trial at Seattle Children’s Hospital.

Otarmeni was granted accelerated approval in cases of severe-to-profound and profound OTOF-related hearing loss based on pivotal results of the CHORD trial demonstrating 80% of participants achieved or surpassed a hearing level meeting the primary endpoint, and with longer follow-up, 42% achieved normal hearing that included whispers.

Read more on the Virginia Merrill Bloedel Hearing Research website, and see the full press release on the Regeneron website.