Regeneron Pharmaceuticals Regeneron CEO Leonard Schleifer told CNBC on Friday that the company plans to make its newly approved gene therapy for a rare genetic hearing loss available for free in the United States, but the company has not yet decided how much it will cost in other countries.
“We haven’t decided on a price yet, but there should be a fair share of the cost outside the U.S.,” Schleifer said in an interview with CNBC’s Becky Quick. “We’re giving it away for free in the US.”
Regeneron announced Thursday that it will make the treatment available for free in the U.S. following a larger agreement with the Trump administration over drug pricing. President Donald Trump has long argued that Americans pay disproportionately more for drugs than other wealthy countries, thereby subsidizing innovation.
Regeneron’s gene therapy Otarmeni treats a rare disease in which a defect in the OTOF gene limits the production of the protein that transmits audio signals in the ear. Otarmeni provides a working copy of the gene. In clinical trials, 16 out of 20 people who received gene therapy, or 80%, experienced improved hearing.
It is estimated that about 50 babies are born with the condition in the United States each year, but because it is such a rare disease, the gene therapy was not expected to be an economic boon for Regeneron. Piper Sandler analysts estimate peak sales at $130 million.
Schleifer said Regeneron decided to make the treatment available for free in the U.S. “to show who we are.”
The treatment was approved under the Food and Drug Administration’s newly created National Priority Voucher Program, which aims to accelerate the review of medicines that align with U.S. national health priorities.
Gene therapy can cost millions of dollars, and European countries have balked at the price tag in the past.
