The most expensive drug in the world costs several million euros

MEDICINES – 3.4 million euros: this is the price for a single dose of Hemgenix treatment. This makes it the most expensive drug in the world to treat hemophilia, and it has just been approved for marketing by US authorities.

Fortunately, this disease remains very rare, but it still affects 400,000 people worldwide and mainly men. Hemophilia is a blood disorder and when a person with this condition bleeds, the blood fails to clot to stop the flow. The consequences can be significant with the risk of fairly severe bleeding.

Currently, people suffering from hemophilia are forced into a background treatment, with daily injections by intravenous infusion. This maintains the missing or affected clotting factor in the patient. But such treatment can seriously deteriorate the quality of life of those affected and this argument has weighed in the balance.

In a statement, the director of the FDA’s Center for Biologics Evaluation and Research said: Despite advances in the treatment of hemophilia, preventing and treating bleeding episodes can have a negative impact on an individual’s quality of life. Today’s approval provides a new treatment option for patients with hemophilia B “.

A new, not-so-expensive option

If the cost of treatment seems exorbitant, a patient with hemophilia in the United States would spend between 21 and 23 million dollars in their lifetime for the current treatment according to a study published in the Journal of Medical Economics in 2021 This is more than double that in Europe, for example, where the salary amounts to 10 million dollars.

3.4 million is therefore immediately cheaper. The product in Hemgenix is ​​based on gene therapy. This method of treatment has already been studied to combat certain genetic diseases such as cystic fibrosis, for example, and it brings real hope in the treatment of genetic diseases. In fact, this makes it possible to deliver to the cells a “healthy” gene capable of correcting a defective gene.

“Gene therapy consists of importing a copy of a functional gene into a target cell, so that it is expressed there and results in the production of the protein that is lacking. The gene is delivered through a vector” reports Inserm. In the case of Hemgenix, the genetic information is then reproduced by the cells, thus spreading this hitherto defective coagulation protein to all the cells. Thus, a single injection is enough and makes it possible to repair the DNA of the damaged gene.

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