The U.S. Food and Drug Administration approved Otarmeni on April 23, 2026, giving accelerated authorization to the gene therapy designed to treat severe-to-profound hearing loss caused by mutations in the OTOF gene [1]. Developed by Regeneron, Otarmeni delivers a functioning copy of the defective gene directly into patients' cells to restore hearing ability [1].
In the pivotal international clinical trial conducted by Mass Eye and Ear and China’s Fudan University, 80 percent of treated patients gained measurable hearing, while 42 percent achieved the ability to hear whispers [1]. Two and a half years after receiving a single injection, 90 percent of patients in the multi-center study maintained hearing [1].
The therapy showed rapid effects in toddlers born deaf. Before treatment, these children showed no response to sound, but weeks after administration, some began to respond to auditory stimuli. Dr. Yilai Shu said, "When the parents realized their child had a response to sound they cried. The whole family cried" [1].
Otarmeni's approval marks a milestone after over two decades of setbacks for gene therapy. The early history was marred by the 1999 death of Jesse Gelsinger in a gene therapy trial, which caused the field to collapse temporarily [1].
The FDA’s accelerated approval allows earlier patient access while Regeneron continues gathering clinical data to confirm the therapy's safety and efficacy over the longer term [1].
