Long occupied by Zolgensma from Norvatis then Skysona from Bluebird, the place of the most expensive drug in the world now goes to Hemgenix. The latter is marketed by the American laboratory CSL Behring and it is used to treat hemophilia B.
Authorized since the end of last November by the powerful Food and Drug Administration (FDA), the drug is available for 3.5 million dollars. While the amount may seem high, it is still much lower than the current cost for a patient over a lifetime estimated between 21 and 23 million dollars in the United States.
A rare disease that prevents blood from clotting
Patients with hemophilia today are forced to undergo lifelong treatment, via daily intravenous infusions. The Hemgenix allows him to be satisfied with a single infusion to treat this rare disease which prevents the blood from clotting properly. Bleeding can occur in joints, muscles or internal organs (including the brain).
According to data reported by the American agency, the most expensive drug in the world would have reduced the expected bleeding rate by 54% over the course of a year. Furthermore, it freed 94% of patients from the long and expensive infusions that are used today to control this life-threatening disease.
The treatment in question is based on the technique of gene therapy: genetic material is introduced into the cells to supplement the diseased gene with a healthy gene. Many scientists have welcomed such FDA approval and point to significant progress in the development of innovative therapies.
1 in 40,000 people worldwide
Hemophilia B affects about one in 40,000 people worldwide, and this version B affects about 15% of all hemophilia patients. In Europe and the United States, it is estimated that approximately 16 million people have hemophilia B. Note that most women who are affected by the disease show almost no symptoms. Hemophilia A would affect 5 times more of the world but it is less restrictive.
