News
Regeneron’s Otarmeni™ Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss
Overview
Approval in severe-to-profound and profound OTOF-related hearing loss is 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.
First FDA-approved example of a gene therapy to restore a neurosensory function to normal levels.
Otarmeni is Regeneron’s first approved genetic medicine, showcasing its ability to advance new therapeutic approaches to address conditions with great unmet medical need.
In This Article
“Otarmeni is a huge scientific leap and is representative of Regeneron’s approaches to continually push the boundaries of science to benefit humanity. 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 U.S. and serves to highlight our belief that the biopharmaceutical industry can be a genuine force for good in the world.”
– George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer of Regeneron.
The Big Picture
Regeneron will provide Otarmeni at no cost to clinically eligible individuals in the U.S. This may not necessarily reflect out-of-pocket costs for administration of this free therapy, which would be outside of the control of Regeneron; individuals should consult with their healthcare provider and/or insurance provider. For more information on access, contact Regeneron’s OnPath with OTARMENI™ patient support program at 1-866-500-GENE (1-866-500-4363).
Otarmeni received Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the FDA.
