Hemophilia is an inherited disorder in which the lack of sufficient blood clotting factor inhibits clotting of blood normally. The clotting factor consists of proteins that wok with platelets to help the blood clot. According to the U.S. Centers for Disease Control and Prevention, hemophilia occurs in around one in 5,000 male births in the U.S. alone. Every year, 400 babies are born with hemophilia in the country. The growing prevalence of hemophilia has led to the growth of the global hemophilia therapeutics market. Technological advancements in hemophilia drug innovation, along with the development of novel coagulating factors, are anticipated to contribute towards the growth of the market in the near future.
The various types of hemophilia include hemophilia A, hemophilia B, and hemophilia C. While hemophilia A is caused due to the deficiency of clotting factor VIII, hemophilia B is caused due to insufficient clotting factor IX. Hemophilia C is caused by the lack of clotting factor XI. Hemophilia A is the most severe form of the disease and is the most common type of disorder.
North America Emerges as Major Market for Hemophilia Therapeutics
Among the key regions in the global hemophilia therapeutics market, North America boasts a high rate of awareness about hemophilia treatment and is the leading region in the market. However, in the coming years, Asia Pacific is anticipated to register robust growth owing to the growing demand from countries such as India and China. Rising medical tourism, a large patient pool, and improving healthcare infrastructure are the key factors contributing towards the growth of the market in this region.
Fitusiran: The Investigational RNAi Therapeutic Drug for Hemophilia
RNA interface, better known as RNAi, refers to a natural process of gene silencing. RNAi therapeutics are a major novel class of medicines created by prompting the natural biological process of RNAi occurring within cells. Alnylam Pharmaceuticals is a biopharmaceutical firm developing novel RNAi therapeutics.
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This month, Alnylam Pharmaceuticals Inc. announced that the company has started administering doses of inhibitors to hemophilia patients in an ongoing clinical trial. The trial evaluates the effect of Fitusiran, an investigational RNAi therapeutic drug, on patients suffering from hemophilia. The drug targets antithrombin (AT) for treating hemophilia A and B, and rare bleeding disorders. To prevent bleeding in patients, Fitusiran lowers AT levels, thereby prompting the generation of sufficient thrombin and formation of fibrin clot to restore hemostasis, the process that inhibits bleeding post injury. The ongoing phase 1 trial of fitusiran is being held across Switzerland, Russia, Bulgaria, and the U.K.
Key Market Players Focussing on Development of Novel Therapeutic Drugs
Some of the key players in the global hemophilia therapeutics market are Genetics Institute, Bayer AG, Novo Nordisk A/S, Pfizer Inc., Baxter International Inc., Biogen Idec Inc., Expression Therapeutics, Alpha Therapeutics Corporation, Swedish Orphan Biovitrum AB, and Expression Therapeutics. The market players are focussing on the development of novel therapeutics for treating hemophilia. The key players are entering into partnerships for research and development as well as commercialization of drugs. For example, Alnylam Pharmaceuticals and Sanofi Genzyme formed an alliance in January 2014 to expand the development and commercialization of RNA therapeutic drugs worldwide.