The United States (US) Food and Drug Administration (FDA) has approved the hemophilia B gene therapy developed by the Australian company, CSL Ltd, making it the most expensive treatment in the world, costing $3.5 million for a one-time treatment.
In comparison to existing medicines from Biogen (BIIB.O), Pfizer (PFE.N), and others, that involve regular infusions, the new gene therapy, Hemgenix (also known as EtranaDez), is the first-ever long-term therapeutic solution for the rare genetic blood clotting condition.
One in every 40,000 persons is infected by the illness, which is characterized by a gene mutation that impairs the body’s capacity to produce factor IX. Currently, the practice of treatment is to inject a concentrated type of clotting factor.
While the new gene therapy uses a modified virus that has a gene that is activated in the liver and produces clotting factor IX.
The company remains confident that this pricing point will result in considerable cost savings for the general healthcare system as well as a significant reduction in the financial impact of hemophilia B.
While the price may have surprised some, it may be justifiable when compared to weekly intravenous injections of factor IX, which cost from $550,000 to $750,000 each year.