This $3.5 million blood-clotting shot has been approved by the FDA
Key Takeaways
The FDA has approved tranacogene dezaparvovec-drlb (Hemgenix) as a single-dose medication for patients with hemophilia B. It is expected to cost $3.5 million per shot.
Research has found that Hemgenix reduced bleeding rates in patients with hemophilia B by 80% compared with factor IX prophylaxis treatment.
There are concerns about Hemgenix’s price tag; a study suggested it would be more cost-effective if priced under $3 million a dose. The drug’s duration has also been questioned.
It may sound like something out of fiction, but a multi-million-dollar medical treatment is a reality—and has been approved by the FDA.
Estimated to cost $3.5 million per dose, the gene therapy medication etranacogene dezaparvovec-drlb (Hemgenix) was approved on November 22, 2022, for administration to adults with hemophilia B, a rare genetic disease that reduces blood clotting.
According to the FDA, Hemgenix (manufactured by CSL Behring LLC) is a one-dose medication intended for patients with hemophilia B who have been using factor IX prophylaxis therapy; experience historical or current life-threatening hemorrhage; or have repeated, serious episodes of spontaneous bleeding.
Hemgenix is “the most expensive drug in the world,” according to an article published by Nature.[] But research shows that it may be cost-effective, even with its hefty price tag.
Efficacy, cost-effectiveness assessed
On November 2, 2022, the Institute for Clinical and Economic Review (ICER) published an evidence report that assessed Hemgenix’s clinical effectiveness and value for treating patients with hemophilia B.[]
ICER researchers assessed the bleeding rates of patients with hemophilia B who received factor IX prophylaxis treatment compared with those who were treated with Hemgenix.
“Patients treated with [Hemgenix] had an 80% reduction in treated joint bleeds and similar reductions in other bleeds when compared with their bleeding rates on factor prophylaxis prior to gene therapy,” the researchers wrote.
The ICER report indicated, however, that there is uncertainty over the duration of Hemgenix’s benefits to patients after its single dose is administered.
ICER suggested Hemgenix would be more cost-effective if priced between $2.93 million to $2.96 million per dose.
Concerns over Hemgenix’s costs could be offset by the savings that are predicted to result from use of the drug. According to Nature, “Hemgenix could save the US health-care system $5 million to $5.8 million per person treated.”
What this means for you
Hemgenix shows promise as an effective treatment for patients with hemophilia B, even in light of its high cost. Time will tell if its manufacturer abides by industry suggestions and reduces the cost. You may want to stay apprised of further research on this medication as its long-term efficacy is assessed.