Regeneron weighs overseas price for Otarmeni, gene therapy for rare type of hearing shortfall
Regeneron will produce its newly approved gene therapy Otarmeni for a rare, genetic form of hearing deficit available for free in the U.S.
The firm hasn’t decided how it will price the therapy overseas, Regeneron CEO Leonard Schleifer told CNBC on Friday. This also touches on aspects of earnings report.
Regeneron proclaimed Otarmeni would be available for free alongside a broader agreement with the Trump administration on drug pricing.
Regeneron Pharmaceuticals will generate its newly approved gene therapy for a rare, genetic form of hearing shortfall available for free in the U.S. The business hasn’t 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 mentioned in an interview with CNBC’s Becky Quick. “In the United States, we’re giving it away for free.”
Regeneron on Thursday published it would create 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 citizens 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 noted Regeneron decided to produce 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.