Hemophilia is a rare condition that affects the blood’s ability to clot. It’s usually inherited. Most people who have it are male. People with hemophilia do not have as many clotting factors as they should have in their blood. This means they bleed for longer than usual.
US regulators approved CSL Behring’s Hemgenix-hemophilia B gene therapy worth $3.5 million a dose, making it the world’s most expensive drug. The drug costs 28.58 crores (28,58,48,675.00) in Indian rupees.
1. Hemgenix works by delivering a gene that can produce the missing clotting factors into the liver, where it starts working to make the Factor IX protein.
2. A study of the therapy found that just a single administration of the drug cuts the number of bleeding events expected over the course of a year by 54%.
3. The drug can also free 94% of patients from time-consuming and costly infusions of Factor IX, which is currently used to control the potentially deadly condition.
4. The gene therapy will be manufactured in Lexington, Massachusetts, by uniQure NV, which sold the commercialization rights for Hemgenix to CSL Behring in 2020.
5. Speaking on whether the costly drug will be a hit, Brad Loncar, a biotechnology investor said “While the price is a little higher than expected, I do think it has a chance of being successful because 1) existing drugs are also very expensive and 2) hemophilia patients constantly live in fear of bleeds. A gene therapy product will be appealing to some.”
Traditional hemophilia treatment infuses missing proteins, called clotting factors, that the body needs to form clots and stop bleeding. Hemgenix works by delivering a gene that can produce the missing clotting factors into the liver, where it starts working to make the Factor IX protein.
The gene therapy will be manufactured in Lexington, Massachusetts, by uniQure NV, which sold the commercialization rights for Hemgenix to CSL Behring in 2020. About 16 million people in the US and Europe have hemophilia B, according to uniQure. Hemophilia A is more common, affecting about five times as many people.
