Regeneron Pharmaceuticals Regeneron CEO Leonard Schleifer told CNBC on Friday that its newly approved gene therapy for a rare, genetic form of hearing loss will be made available for free in the US. The company has not yet decided how much it will charge in other countries.
“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’re giving it away for free.”
Regeneron announced Thursday that it will make the therapy available for free in the U.S., along with a major deal with the Trump administration regarding drug pricing. President Donald Trump has argued for years that Americans are unfairly paying more for drugs than other wealthy countries and, as a result, are subsidizing innovation.
Regeneron’s gene therapy Otarmeni treats a rare disease where a faulty OTOF gene limits the production of a protein that transmits sound signals to the ear. Otarmeni provides a working copy of the gene. In one clinical trial, 16 out of 20 or 80% of people who received gene therapy experienced improvement in their hearing.
It is estimated that about 50 babies are born with the condition each year in the U.S. Because it is so rare, the gene therapy was not expected to be a financial boon for Regeneron. Piper Sandler analysts estimate peak sales of $130 million.
Schleifer said Regeneron decided to make the treatment available for free in the US “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 review of drugs in line with US national health priorities.
Gene therapy can cost millions of dollars, and European countries have balked at the price tag in the past.
