Regeneron weighs overseas price for rare hearing loss gene therapy
Regeneron Pharmaceuticals Regeneron will make its newly approved gene therapy for a rare genetic form of hearing loss available for free in the United States, CEO Leonard Schleifer told CNBC on Friday. He said the company hasn’t 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 U.S. as well,” Schleifer said in an interview with CNBC’s Becky Quick. “We give it away for free in the United States.”
Regeneron announced Thursday that it will offer the therapy for free in the United States, along with a larger agreement on drug pricing with the Trump administration. President Donald Trump has argued for years that Americans unfairly pay more for drugs than other wealthy nations, spurring innovation as a result.
Regeneron’s gene therapy Otarmeni treats a rare disease in which a faulty OTOF gene limits production of a protein that transmits sound signals in the ear. Otarmeni delivers a working copy of the gene. In one clinical trial, 16 out of 20 people who received gene therapy experienced improvement in their hearing, or 80%.
It is estimated that approximately 50 babies a year are born with this disease in the United States. Because it is such a rare condition, the gene therapy was not expected to be a financial boon for Regeneron. Piper Sandler analysts predicted peak sales of $130 million.
Regeneron decided to offer the treatment for free in the U.S. “to show who we are,” Schleifer said.
The treatment was approved under the Food and Drug Administration’s newly created National Priority Voucher program, which aims to expedite reviews of drugs that align with U.S. national health priorities.
Gene therapies can cost millions of dollars, and European countries have opposed price tags in the past.
