A cure for the blood-clotting disorder hemophilia B is listed at a cost for $3.5 million per dose, as reported by the Food and Drug Administration.
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The U.S. Food and Drug Administration approved Hemgenix last Tuesday, a gene-therapy treatment to help adults with hemophilia B, a genetic disorder. The disorder prevents people from producing a specific protein to clot blood when bleeding occurs. Previously, hemophilia treatments required routine injections for patients in order to maintain adequate levels of the missing protein as reported by Oddity Central. Hemgenix only requires one single IV infusion for an individual to be cured forever but at a cost.
”This historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for years after a one-time infusion. With the approval of HEMGENIX, CSL now offers an even more comprehensive portfolio of treatments for people living with hemophilia B, ushering in a new era of treatment options.”
Apparently, the cure costs $3.5 million per dose which makes it the most expensive drug in the world. The product was first developed by Dutch biotechnology company UniQuire and CSL Behring paid $450 million for the licensing. Additionally, the company sells two approved Factor IX therapies for hemophilia B.
CSL CEO and Managing Director Peter Perrault said the new breakthrough will help people living with hemophilia B and the company promises to bring new breakthrough medications in the future.
”As part of our promise to patients, CSL is committed to delivering innovative and groundbreaking solutions to address unmet medical needs, and we are proud to introduce the next wave of breakthrough medicines for people living with hemophilia B. We recognize and thank all trial participants, scientists and investigators—without whom this important achievement would not have been possible—and look forward to seeing the positive impact of HEMGENIX on the hemophilia B community.”
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The Institute for Clinical and Economic Review determined that the price of Hemgenix would be priced at $2.9 million, deeming it to be “fair” for its cost. However, the high price tag will deter patients since hemophilia B affects about 6,000 people in the United States and only 15 percent will be eligible for the drug. In a report by Forbes, the expected drug sales will be around $1.2 billion.
Despite the high price tag for the newly approved gene therapy for hemophilia B, Hemgenix, the budget impact for payers will be manageable. It’s other gene therapies – in pipeline and indication expansion of approved products – that are more of a concern.https://t.co/4g0I7DWp7g
— Joshua P. Cohen (@JoshuaPCohen1) December 2, 2022
During the trial, CSL revealed in their press release that the symptoms of HEMGENIX include flu-like symptoms, fatigues, nausea, liver enzyme elevations, and infusion-related actions. However, doctors will monitor the reactions for at least three hours after administration. Patients are encouraged to submit any negative side effects to the FDA.