The Federal Drug Administrations(FDAfor its acronym in English) of the United States approved a new treatment for a rare disease of the blood clottingbut for its price, it is classified as the most expensive in the world: per dose it will cost 3.5 million dollars, something like 67 million pesos.
The information was published on the site Science Alert where it is detailed that the medicine, called Hemgenixis a treatment of gene therapy for the hemophilia Bwhich is a rare genetic disease that causes reduced blood clotting blood. The most serious symptoms include spontaneous and repeated bleeding episodes that are difficult to stop.
Although the cost can be very high, a recent analysis of the cost-effectiveness of the drug suggests that it is a relatively “fair” price for what the treatment accomplishes, at least in the United States.
“The approval provides a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies for those who experience a high burden of disease associated with this form of hemophiliasaid Peter Marks, director of the FDA’s Center for Biological Research and Evaluation.
How does Hemgenix work?
Hemgenix it is an intravenous product that is administered in a single dose at a fraction of the price. The drug is transported into the body via a virus-based vector, which is designed to deliver dna to target cells in the liver. This genetic information is then replicated by the cells, disseminating the instructions for a clotting protein, known as Factor IX.
Two studies have so far tested the efficacy and safety of Hemgenix. In a study among 54 participants with hemophilia B severe or moderately severe, the researchers found higher levels of activity of the Factor IXreducing the need for routine replacement therapies currently available to patients.
The previous record holder for the most expensive drug was another form of “one shot” gene therapy to treat the spinal muscular atrophy has an estimated cost of 2 millions of dollarsmore than 38 million pesos.