(CNN) — The US Food and Drug Administration (FDA) on Tuesday approved Hemgenix, a new drug to treat hemophilia. Drugmaker CSL Behring set the price at $3.5 million per treatment, making it the most expensive drug in the world.
Hemgenix is a gene therapy to treat adults with hemophilia B, a genetic bleeding disorder in which patients do not produce a protein needed to create blood clots. About one in 40,000 people have the disease, most of whom are men.
Typical treatment requires routine intravenous infusions to maintain the missing or deficient clotting factor at high levels. However, the new gene therapy is intended to be a single IV infusion.
“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advances in the treatment of hemophilia, the prevention and treatment of bleeding episodes can have a negative impact on people’s quality of life,” Dr. Peter Marks, director of the Center for Hemophilia Evaluation, said in a statement. and FDA Biological Research. “Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those experiencing the disease associated with this form 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 to CNN that Hemgenix is now the most expensive drug in the world.
In a recent analysis of the drug’s profitability, which weighed health benefits against compensation costs, ICER suggested that a fair price for the drug would be between $2.93 and $2.96 million.
According to a GoodRx analysis, the most expensive drug in the US so far was Zolgensma, which was approved by the FDA in 2019 to treat spinal muscular atrophy and was priced at $2.1 million per treatment.
The FDA approved Hemgenix based on safety and efficacy evaluated in two studies of approximately 60 adult men.