Regeneron to Provide Rare Hearing Loss Gene Therapy Free of Charge in U.S.

Regeneron Pharmaceuticals has announced that Otarmeni, a gene therapy targeting a rare genetic form of hearing loss, will be available at no cost to patients in the U.S. CEO Leonard Schleifer confirmed the decision, noting that while the U.S. market will be free, international payers will be expected to contribute their fair share. The move coincides with a broader agreement between Regeneron and the Trump administration regarding drug pricing. The administration has long contended that U.S. consumers disproportionately subsidize global medical innovation by paying higher prices than other developed nations. Otarmeni treats a condition caused by a faulty OTOF gene, which impairs the production of proteins necessary for transmitting sound signals. In clinical trials, the therapy demonstrated an 80% success rate, with 16 out of 20 participants showing hearing improvements. The therapy was approved via the FDA's National Priority Voucher program, designed to accelerate reviews for drugs aligning with national health priorities. Given the rarity of the condition—affecting approximately 50 infants annually in the U.S.—the drug is not expected to be a primary revenue driver. Analysts at Piper Sandler have estimated peak sales for the treatment at $130 million. While the U.S. waiver limits immediate domestic revenue, the move serves as a strategic alignment with federal health priorities. The company now faces the challenge of pricing the therapy in European markets, where high costs for gene therapies have previously met significant resistance.