For adults with Hemophilia B, the FDA has approved a $3.5 million gene therapy.
American health officials approved the first Hemophilia gene therapy, a $3.5 million one-time treatment for the blood-clotting condition. The Food and Drug Administration has approved Hemgenix, an IV therapy for persons with haemophilia B, a less common form of the hereditary condition that primarily affects men. The drug's maker, CSL Behring, revealed the $3.5 million price tag as soon as the FDA gave its approval, arguing that because fewer bleeding episodes and clotting therapies would be needed as a result of the medication, overall healthcare costs would be lower. The cost appeared to be higher than the cost of several other gene therapies that cost more than $2 million. This approval offers patients with hemophilia B a new treatment option and marks significant advancement in the creation of cutting-edge treatments. How long the treatment lasts was not made clear by the organization. Mutations in the gene for a protein required for blood clotting are what give rise to hemophilia, which nearly exclusively affects men. Many patients require treatments once or more a week to stop significant bleeding from small cuts or bruises since they can be life-threatening. If the illness is not addressed, bleeding may develop that seeps into the brain and other organs, including the joints. Hemgenix provides a functioning gene to the liver, which makes the clotting protein. According to the FDA, hemophilia B affects approximately 1 in 40,000 individuals and accounts for 15% of those who have the condition. According to the FDA, it approved the medicine based on two small trials, including one that revealed people using it had higher levels of the clotting protein, needed less conventional care, and experienced a 54% decrease in bleeding issues. A comparable gene treatment for hemophilia A was approved by European regulators earlier this year. FDA review of that medicine, manufactured by BioMarin, is currently ongoing.
Reference: - https://www.bmj.com/content/379/bmj.o2858#:~:text=Marketed%20as%20Hemgenix%2C%20etranacogene%20dezaparvovec,single%20dose%20by%20intravenous%20infusion.