Orphan blood disease drugs to gain market

Updated on 7 February 2013

Jakafi (ruxolitinib) is a JAK inhibitor which works to target the genes believed to cause MF, approved by the FDA in November 2011 for the treatment of intermediate or high-risk MF, and is perhaps set to be a future blockbuster drug. Kimberley Carter, Associate Analyst at GBI Research states: "The exceptional performance of Soliris has shown that there is a great opportunity for pharma companies in the development of novel, curative drugs for orphan blood disorders. Many patients with these diseases rely on generic drugs that often come with debilitating side effects and may require a high level of commitment from the patient, such as regular hospital treatments. Therefore, the unmet needs of these patients are very high and the right therapy would perform very well."

Other drugs with the same mechanism of action are currently in the development pipeline, as other pharmaceutical companies fight to compete for Jakafi's market share, and as a result of this healthy competition, the market for pharmaceutical treatment for MF is expected to grow at a CAGR of 34% throughout 2011-2019 to reach a value of US$1,127m by 2019.

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