FDA Approves Most Expensive Drug in the World

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An IV drip
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On Nov. 22, 2022, the FDA approved Hemgenix, which treats hemophilia B in adults. The drug will cost $3.5 million per treatment, set by manufacturer CSL Behring, making it the most expensive drug in the world, according to Institute for Clinical and Economic Review (ICER).

The drug is about 66.7% more expensive than Zolgensma, which was previously the most expensive drug in the world at $2.1 million per treatment course. Zolgensma was approved by the FDA in 2019 for the treatment of spinal muscular atrophy.

Hemgenix is the first gene therapy (specifically an adeno-associated virus vector-based gene therapy) for hemophilia B. According to the FDA, “Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding. Symptoms can include prolonged or heavy bleeding after an injury, surgery, or dental procedure; in severe cases, bleeding episodes can occur spontaneously without a clear cause. Prolonged bleeding episodes can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain.”

Those with hemophilia B, typically men, generally have to have routine prophylactic IV (intravenous) infusions of Factor IX replacements so the body can maintain “sufficient levels of clotting factor to prevent bleeding episodes.” The FDA noted that Hemgenix is a one-time therapy given by IV.

Because Hemgenix should eliminate the need for more routine prophylactic IV infusions and, thus, eliminate the cost of those treatments, ICER considers the price cost-effective. Estimates for life-long adult prophylactic treatment of hemophilia B can range from $21 to $23 million. For post-bleed treatments, patients pay an estimated $22 million for adult treatments.

ICER stated that a “fair price” for Hemgenix was somewhere between $2.93 million and $2.96 million per treatment due to the cost-effectiveness over prophylactic treatments. However, ICER was careful to note that the “prophylactic treatment… itself is widely considered to be far too expensive.”

Discussion Questions

1. Should the US government regulate prescription drug prices? Why or why not?

2. Should some drugs, such as Hemgenix, that replace a lifetime of other drugs be allowed to be so costly? Explain your answer(s).

3. Should drug companies be allowed to set drug prices without oversight? Why or why not?


Carly Cassella, “The FDA Just Approved The Most Expensive Drug in The World,” sciencealert.com, Nov. 24, 2022

FDA, “FDA Approves First Gene Therapy to Treat Adults with Hemophilia B,” fda.gov, Nov. 22, 2022

ICER, “ICER Publishes Evidence Report on Gene Therapies for Hemophilia A and B,” icer.org, Nov. 2, 2022

Nanxin Li, et al., “Adult Lifetime Cost of Hemophilia B Management in the US: Payer and Societal Perspectives from a Decision Analytic Model,” Journal of Medical Economics, tandfonline.com, Dec. 21, 2020
Deidre McPhillips, “FDA Approves $3.5 Million Treatment for Hemophilia, Now the Most Expensive Drug in the World,” cnn.com, Nov. 23, 2022