This is the most expensive drug in the world
Approved by the FDA
The FDA has approved a new treatment for hemophilia, which is the most expensive in the world since each dose costs $3.5 million! The name of the drug is Hemgenix and it is a gene therapy for hemophilia B, a rare genetic disease that causes impaired blood clotting. In severe form, it can be life-threatening since any bleeding is difficult to stop. It’s more common in men, and while it’s hard to estimate how many suffer from it, an estimated 8,000 men in the US have it.
Current treatment involves a clotting factor that patients must take for life, with the total cost being enormous, reaching up to $23 million per patient over their lifetime.
Hemgenix offers a single dose. It enters the body and provides DNA to target liver cells. The genetic information is then reproduced by the cells, containing instructions for the formation of a clotting protein known as Factor IX.
Two separate studies have proven the drug’s effectiveness, with patients experiencing uncontrolled bleeding reduced by 50%.
Gene therapy for hemophilia has been on the horizon for two decades. Despite the advances in the treatment of the disease, the prevention and treatment of bleeding episodes have serious effects on the quality of life of patients. Today’s approval offers a new treatment option for patients with hemophilia B and represents significant progress in the development of innovative treatments for those facing a severe form of this disease.
It is not yet clear whether patients can completely get rid of hemophilia, but the initial results are promising. The European Medicines Agency is also looking into gene therapy and it will be interesting to see how much Hemgenix will cost in the Old Continent.
