U.S. FDA approves first gene therapy for inherited deafness, offered free to U.S. patients
Gene therapy can now restore hearing to children born deaf — and Regeneron is giving it away free to U.S. families. In a trial of 20 children with rare OTOF mutations, 16 gained meaningful hearing within about five months, and several were brought to essentially normal hearing. One toddler covered his ears when an ambulance siren passed — his first sign of sound. Instead of charging up to $4 million per child, as is common for rare-disease therapies, Regeneron chose a different path entirely. Beyond the families it directly helps, the decision hints at a quietly radical idea: that breakthrough medicine for rare conditions doesn’t have to come with a breathtaking price tag.









