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The US Food and Drug Administration (FDA) has just given its marketing authorization to a drug worth 3.5 million dollars per dose. Hemgenix is a gene therapy that overcomes the genetic deficit of hemophilia B.
It is often said that health is priceless. For patients with hemophilia B, she might have one: $3.5 million. This is the price of the dose of the most expensive drug in the world, marketed by the CSL Behring laboratory, called Hemgenix. It has just obtained marketing authorization from the American Food and Drug Administration.
What is hemophilia B?
People who suffer from hemophilia B are affected by a genetic mutation. This causes them to have an inherited coagulation factor IX deficiency, which prevents their blood from clotting properly.
For these patients, the risk of hemorrhage in the joints, muscles or even internal organs is very high. Affecting one in 40,000 people, haemophilia B accounts for 15% of haemophilia patients worldwide. In hemophilia, there are more patients affected by form A of the disease, on average five times more than form B.
Only one dose of treatment to take
For these patients, a substitution treatment exists. It temporarily compensates for the coagulation factor deficiency. Administered intravenously, it must be taken for life to avoid bleeding. This can greatly impact the quality of life of patients.
The difference with Hemgenix is that the patient will only need to take a single dose of treatment, which will be enough for life.
This drug consists of a viral vector carrying a gene for coagulation factor IX, a gene therapy based on an adenovirus. The latter is used as a vector to deliver genetic material, a portion of DNA, into a human cell.
Everything then happens in the patient’s liver. The clotting factor IX gene is delivered to this organ, so that it can start producing the previously missing factor IX protein. The treatment then makes it possible to increase blood levels of factor IX and to limit bleeding episodes.
According to clinical studies, 94% of patients would be able to do without regular infusions of conventional treatment and Hemgenix would reduce by half the number of bleeding events likely to occur over the course of a year, in patients.
Why such a price?
If the drug seems really revolutionary for patients, it also remains unaffordable for the majority of them. For the manufacturing laboratory, CSL Behring, “this award will generate cost savings for healthcare systems and significantly reduce the burden of hemophilia B.”, believing that a single dose will ultimately cost less than current lifelong treatment of sufferers. It remains to be seen at what price it will be set by the authorities in France.