America’s drug regulator FDA has approved the world’s most expensive drug. The B gene therapy drug, called HemGenix, is designed to treat CSL Behring’s hemophilia, a rare blood disorder. It has been approved for marketing by the Food and Drug Administration. The cost of one dose of this is 35 lakh dollars (about 28.62 crore rupees).
The patient of hemophilia gets a wound on the body and the blood keeps flowing from the wound. No matter how much time passes, it does not stop. If the flow of blood continues, the patient will die due to lack of blood in the body. Such a patient is given an injection of a protein called Factor-9 to stop the bleeding. This treatment has to be done at regular intervals over a long period of time. A major study of this expensive drug showed that patients taking a single dose of Hemgenics experienced a 54 percent reduction in the incidence of fatal bleeding over one year.
What happens in hemophilia?
If the patient of this disease gets any injury and starts bleeding, then it does not stop. We all have a special gene protein called Protein-S in our blood. When bleeding occurs, the blood coming out of the wound coagulates with proteins when exposed to air and stops the flow of blood. This protein is not present in the blood of a hemophilia patient. That’s why the blood does not clot when there is a wound, it keeps on flowing continuously.
most expensive drug ever
Novartis’ drug was approved in 2019 for pediatric spinal muscular atrophy. Then its price was 2.1 million dollars. Similarly, the cost of another blood disorder medicine was $2.8 million. It has been approved this year itself.