On Tuesday, the Food and Drug Administration approved the most expensive drug ever, with a price tag of $3.5 million.
CSL Behring’s Hemgenix is the first FDA-approved gene therapy for the treatment of hemophilia B, a bleeding illness caused by a lack of a protein called Factor IX. In a new multimillion-dollar gene treatment for severe hemophilia B, the gene for the blood-clotting factor IX is infused intravenously (IV) in a single dose. Consequently, injections are no longer required on a regular basis.
According to Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research, “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.”
This bleeding disorder affects about 1 in 40,000 people; men are more likely to be affected than women. An injury, surgery, or dental work can cause severe or prolonged bleeding in those with hemophilia B. People with hemophilia B are at high risk for internal bleeding, especially in the brain, joints, muscles, and other organs.
The CEO of a biotech company, Brad Loncar, told Bloomberg that despite the drug’s higher price tag, he believes it has a chance of success because 1) existing treatments are also quite pricey and 2) hemophilia sufferers continually live in fear of bleeds. In some cases, a person may benefit from gene therapy.
Hemgenix inserts the gene for clotting factor IX into cells through a virus. According to the Food and Drug Administration, lowering both the frequency and severity of bleeding episodes can be achieved by increasing the expression of genes in the liver, which in turn increases the synthesis of the Factor IX protein.
With “two investigations of 57 adult men 18 to 75 years of age with severe or moderately severe Hemophilia B,” the FDA authorized a drug that had been in development for over two decades.
According to the Institute for Clinical and Economic Review and CNN, the annual price tag for Hemgenix is a staggering $3.5 million. Because of this, it is the most costly pharmaceutical product in the world. The price of CSL Behring’s drug was made public shortly after the company received FDA approval. When comparing cost per dose, Hemgenix’s SMA gene therapy is $1 million less than Novartis’ Zolgensma.