3.5 million dollars a dose, just that. This is the record price of the most expensive new drug in the world, which has just been approved for sale in the United States.
- It is not the first time that a new drug has reached such exorbitant prices. In 2019, Novartis’ Zolgensma, for babies with spinal muscular atrophy, was set at $2.1 million.
- The treatment from the biotechnology company Bluebird Bio, formulated against beta-thalassemia, a blood disease, had been set at $2.8 million.
A whole life paid at the average salary in France (2,340 euros net per month) would not even be enough to offer it to you. Hemgenix, a drug for patients with hemophilia B, marketed by the CSL Behring laboratory, has just broken the record for the most expensive drug in the world. The Food and drug administration (FDA), the authority that authorizes the marketing of drugs in the United States, has approved its marketing. Its price: 3.5 million dollars per dose.
Drug could help millions of hemophilia B patients
Hemophilia B is a genetic mutation that causes a hereditary deficiency preventing the blood from clotting properly, which can cause bleeding in the muscles, joints or even internal organs. 1 person in approximately 40,000 suffers from hemophilia B worldwide, which corresponds to 15% of hemophilia patients. In the United States and Europe, it is estimated that about 16 million patients have hemophilia B, while the less rare hemophilia A affects five times as many.
“Many women with the disease have no symptoms. However, 10 to 25% of affected women have mild symptoms; in rare cases, the symptoms are moderate or even severe”, says the FDA. Apart from this expensive drug, there is already an alternative treatment administered intravenously which temporarily compensates for the genetic deficit and facilitates coagulation, which patients must follow throughout their life in order to avoid bleeding.
Why is this treatment so expensive?
This is where the Hemgenix makes the difference: this treatment is a gene therapy that requires only one dose for life. How ? Thanks to an adenovirus capable of providing genetic material, a portion of DNA, to the cells, in order to compensate for the genetic mutation and restore the blood’s ability to clot. The drug would thus make it possible to reduce the number of hemorrhages per year by more than 50% and 94% of patients could finally do without all the infusions necessary to control the disease, according to clinical studies carried out.
But how did the laboratory justify such an exorbitant price? “We believe this award will generate cost savings for healthcare systems and significantly reduce the burden of haemophilia B”argues CSL Behring in a communicated. According to them, the cost of a single dose would be much lower than the total cost of the treatment that hemophiliacs B took for life until now. An argument far from convincing the medical world, which hopes that the French health authorities will manage to negotiate the price downwards.
