The US Food and Drug Administration (FDA) approved the world’s most expensive drug on Tuesday.
Thus, the agency gave the green light to the drug Hemgenix from biopharmaceutical company CSL Behring, which set its price at $3.5 million a dose shortly following receiving FDA approval, making it the most expensive treatment in the world.
Hemgenix is a one-time intravenous medicine given to adult patients with hemophilia B, a bleeding disorder that affects mostly men and is due to a genetic mutation of a protein needed for blood clotting. If left untreated, the condition can cause bleeding to leak into organs and joints, including the brain.
The medicine consists of a viral vector that carries a coagulation factor IX gene that is expressed in the liver in order to increase the levels of this enzyme in the blood and thus limit bleeding episodes.
“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 experiencing a high burden of disease associated with this form of hemophilia,” said Peter Marks, Director of the Evaluation Center. and FDA Investigation.