Hemgenix given to treat first hemophilia B patient in US
One-time gene therapy, approved by FDA in 2022, carries list price of $3.5M
A first adult with hemophilia B in the U.S. has been treated with commercially available Hemgenix (etranacogene dezaparvovec), a recently approved and first gene therapy for the disease, CSL Behring announced.
“As part of our longtime promise to patients, CSL Behring is thrilled to mark this latest milestone of the first commercial patient having received Hemgenix and look[s] forward to continuing to deliver this paradigm-shifting treatment to the hemophilia B community,” Bob Lojewski, CSL Behring’s senior vice president and general manager, North America, said in a company press release.
The company acquired rights to market Hemgenix from uniQure, which originally developed the therapy, in 2021.
Hemgenix delivers healthy version of F9 gene to liver cells
“To the advocacy organizations, medical experts … and the entire hemophilia community who have collaborated with us to establish access for this innovative therapy, we thank you and look forward to many more patients benefiting from this therapy,” Lojewski said.
Hemophilia B is caused by mutations in the F9 gene that provides instructions for making the clotting protein factor IX, known as FIX. Hemgenix is designed to deliver a healthy version of this gene to cells in the liver where most clotting proteins are made.
Hemgenix became the first gene therapy approved by the U.S. Food and Drug Administration to treat adults with moderate-to-severe hemophilia B in November 2022. It was approved in the European Union in February.
Both approvals were supported by data from the ongoing Phase 3 HOPE-B trial (NCT03569891), which tested Hemgenix in 54 men with hemophilia B. Results showed that one-time treatment with Hemgenix led to a substantial reduction in bleeding rates, with most patients being able to discontinue standard preventive bleeding treatments.
The trial is due to conclude in March 2025.
“Hemgenix is an important addition to the current treatment paradigm and a long-awaited advancement for the hemophilia B community,” said Matthew Ryan, MD, a hematologist and medical director at the Hemophilia Outreach Center in Green Bay, Wisconsin.
“This exciting treatment offers patients a chance to live a life without frequent bleeds or the burden of regular infusions. The availability of this new one-time treatment also sparks additional conversations in the community when it comes to treatment options and goals, which is extremely important when managing a life-long condition, as an individual’s needs can change for a variety of reasons,” Ryan said.
Insurers setting up ‘clear policies’ regarding costly therapy’s use
CSL Behring set the U.S. list price for Hemgenix at $3.5 million. Since it aims to reduce bleeding rates and the use of other therapies, the company proposes that Hemgenix offers cost savings to the healthcare system at large. It notes that the typical hemophilia B patient accrues more than $20 million in medical costs throughout adulthood.
At present, health insurance providers covering nearly two-thirds of the U.S. population have established “clear” policies regarding if and how the gene therapy will be covered, CSL Behring stated.
“Discussions between CSL Behring and the payer community on the value proposition offered for Hemgenix have been positive, which is reflected in the policies that have been written,” Lojewski said. “As of May 2023, payers covering roughly 60 percent of the U.S. population have established clear medical policies covering Hemgenix. For those who have not yet established policies, we anticipate that coverage decisions for Hemgenix will be reviewed on a case-by-case basis.”
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