On November 22, the Food and drug administration (Fda), a US regulatory body, approved the most expensive medicin in the world, a therapy to treat hemophilia B, a rare genetic disease.
Hemgenix, this is the name of the medicine produced by the pharmaceutical company Csl Behring, consists of a gene therapy and will cost $ 3.5 million per dose, which, as Bloombergreports, makes it the most expensive drug in the world.
According to clinical studies conducted so far, Hemgenix would be able to eliminate, in more than half of the patients tested, the bleeding episodes associated with the disease, with a potential saving on health costs.
In Europe, the medicine is still being evaluated by the European Medicines Agency (EMA).
On November 22, the Food and drug administration (Fda), a US regulatory body, approved the most expensive medicin in the world, a therapy to treat hemophilia B, a rare genetic disease. Hemgenix, this is the name of the drug produced by the pharmaceutical company Csl Behring, consists of a gene therapy and will cost $ 3.5 million per dose, which, as Bloombergreports, makes it the most expensive medicin in the world. According to clinical studies conducted so far, Hemgenix would be able to eliminate, in more than half of the patients tested, the bleeding episodes associated with the disease, with a potential saving on healthcare costs.
Hemophilia B is a rare genetic disorder caused by the lack of a blood clotting factor, a protein necessary to stop bleeding in case of injuries; as reported by the Higher Institute of Health (ISS), hemophilia B concerns one case in 30,000 in the general population. Being a disease associated with the sex chromosome X, most people who experience symptoms are men, while generally women are healthy carriers. The treatment of this disease generally involves the administration of the missing clotting factor for preventive purposes, or on request, to stop any bleeding.
Hemgenix, however, works differently: it is an adenoviral vector administered intravenously in a single dose, which carries the gene for the missing coagulation factor; Once in circulation, the gene is expressed in the liver, where it produces the protein needed to prevent bleeding episodes.
The safety and efficacy of Hemgenix were evaluated in two clinical studies involving 57 men with severe or moderately severe haemophilia B: in one of the studies, participants experienced increased levels of missing clotting factor activity (resulting in less need for preventive treatment) and a 54% reduction in the rate of annual bleeding events.
The innovativeness of the new drug has a cost, which, in this case, is decidedly high: according to Bloomberg, in fact, it would cost Hemgenix 3.5 million dollars per dose. Instead, a study by Institute for clinical and economic review, a nonprofit organization that deals with the costs of medicines, reports that a fair price for Hemgenix would be between $2.93 million and $2.96 million per dose.