The Unites States and Drug Administration on Tuesday approved Hemgenix for treatment of hemophilia.
The Institute for Clinical and Economic Review, an independent nonprofit research organization that analyzes the value of prescription drugs and other medical products, confirmed for CNN that Hemgenix is now the most expensive drug in the world, with the price at 3.5 million dollars per treatment.
The manufacturer CSL Behring confirmed that the Hemgenix drug is a gene therapy to treat adults with hemophilia B.
Hemophilia B is characterized by lack of the protein responsible for creating blood clots.
Statistics show that about 1 in every 40,000 people, mostly men, have the disease.
Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement that usual treatments require routine intravenous infusions to maintain sufficient levels of the missing or deficient clotting factor, but the new gene therapy is meant to be a one-time IV infusion.
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“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,”
“Today’s approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.” Dr. Mark stated.
FDA approved Hemgenix based on the two studies conducted on about 60 adult men.
Side effects included high liver enzymes, headaches, flu-like symptoms, and mild infusion-related reactions.