Regeneron weighs overseas price for rare hearing loss gene therapy
“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 on Thursday announced it would make the therapy available for free in the U.S. alongside a larger deal with the Trump administration around drug pricing. President Donald Trump, for years, has argued that Americans are unfairly paying more for drugs than other wealthy nations and, as a result, is subsidizing innovation.
Regeneron’s gene therapy Otarmeni treats a rare disease where 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 a clinical trial, 16 of 20, or 80%, of people who received the gene therapy experienced improvements to their hearing.
About 50 babies a year are estimated to be born with the condition in the U.S. Because it’s so rare, the gene therapy wasn’t expected to be a financial boon for Regeneron. Piper Sandler analysts estimated peak sales of $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 reviews of drugs that align with U.S. national health priorities.
Gene therapies can cost millions of dollars, and European countries have balked at the price tags in the past.
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