Regeneron weighs overseas price for gene therapy for rare hearing loss
Regeneron Pharmaceuticals will make its newly approved gene therapy for a rare genetic form of hearing loss available free of charge in the United States. The company has not yet decided how much it will charge in other countries, Regeneron CEO Leonard Schleifer told CNBC on Friday.
“We haven’t set a price yet, but they should pay their fair share outside the United States,” Schleifer said in an interview with CNBC’s Becky Quick. “In the United States, we give it away for free.”
Regeneron announced Thursday that it would make the therapy available free of charge in the U.S., along with a larger deal with the Trump administration on drug pricing. President Donald Trump has argued for years that Americans unfairly pay more for drugs than other wealthy nations and that he is therefore subsidizing innovation.
Regeneron’s gene therapy Otarmeni treats a rare disease in which a faulty OTOF gene limits the production of a protein that transmits sound signals in the ear. Otarmeni provides a working copy of the gene. In a clinical trial, 16 out of 20, or 80%, of people who received the gene therapy saw an improvement in their hearing.
It is estimated that approximately 50 babies are born with this condition each year in the United States. Because the disease is so rare, gene therapy was not expected to be a financial boon for Regeneron. Analysts at Piper Sandler estimated peak sales at $130 million.
Schleifer said Regeneron decided to make the treatment available free of charge 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 expedite the review of medications that align with U.S. national health priorities.
Gene therapies can cost millions of dollars, and European countries have historically balked at the price tags.
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