Alnylam Reviews Impact of Fitusiran on Hemophilia Patients


Published Date : Mar 16, 2016

RNA interface, better known as RNAi, refers to a natural process of gene silencing. RNAi therapeutics is 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. 

This month, Alnylam Pharmaceuticals Inc. has 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 level, thereby prompting 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 is being held across Switzerland, Russia, Bulgaria, and the U.K. 

Hemophilia Therapeutics to Bolster across Asia Pacific
Among the key regions in the global hemophilia therapeutics market, North America boasts of high rate of awareness about hemophilia treatment and is the leading region in the market. 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.  

However, in the coming years, Asia Pacific is anticipated to register a 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. Anticipating the robust growth of the market, 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.