Regeneron Pharmaceuticals will make its recently approved gene therapy for a rare genetic form of hearing loss available for free in the U.S. 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 for free in the U.S. along with a broader agreement with the Trump administration on drug pricing. President Donald Trump has argued for years that Americans are unfairly paying more for medicines than other wealthy nations and are subsidizing innovation as a result.
Regeneron's Otarmeni gene therapy treats a rare disease in which a defective OTOF gene limits the production of a protein that transmits sound signals in the ear. Otarmeni delivers a functional copy of the gene. In a clinical trial, 16 out of 20, or 80%, of people who received gene therapy experienced improvements in their hearing.
It is estimated that about 50 babies a year are born with this condition in the U.S. Because it is so rare, the gene therapy was not expected to be a big financial boon for Regeneron. Analysts at Piper Sandler estimated peak sales at $130 million.
Schleifer said Regeneron decided to make the treatment available for free in the United States, “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 reviews of drugs that align with U.S. national health priorities.
Gene therapies can cost millions of dollars and European countries have objected to the prices in the past.
