This Tuesday, November 22, 2022, the Food and Drug Administration (FDA) authorized the marketing of a new drug on American soil: Hemgenix®, produced by the American laboratory CSL Behring.
Hemgenix® was developed to combat hemophilia B, a rare hematological disease (it affects around 1 in 40,000 people) which is characterized by heavy and/or prolonged bleeding. These can occur in the event of trauma (a cut, for example) but also spontaneously (without an identified cause, therefore).
Extremely expensive gene therapy
People who suffer from hemophilia B have a coagulation defect, linked to the deficiency of a specific protein – factor IX. In the most severe cases, patients should receive regular factor IX infusions.
Hemgenix® (which also comes in the form of an infusion) uses gene therapy: this treatment acts directly at the source of the disease by causing a replacement of “sick” genes by “healthy” genes in patients. This innovative treatment has been tested with 57 patients suffering from haemophilia B.
Yes, but here it is: the Hemgenix® has a particularity that makes it difficult for American patients to access. This treatment is indeed extremely expensive since a single dose costs 3.5 million dollars, or 3.4 million euros: it is therefore the most expensive drug in the world to date. Even so, the FDA believes that Hemgenix® “brings a new treatment option to patients with hemophilia B“.
Source : Food and Drug Administration (FDA)
