Shire Wins Key Approval for Rare Disease Drug

NEC member Shire plc has received approval from the U.S. Food and Drug Administration (FDA) for Takhzyro, a new medication to treat a potentially life-threatening rare disease.  Takhyzro is a first-of-its-kind preventive treatment for a genetic disease called hereditary angioedema, or HAE, which leads to episodes of swelling in the limbs, abdomen and airway.

Shire’s studies have found that patients who take the injectable drug every two weeks experience an 87 percent reduction in attacks.  The addition of this new medication to Shire’s line-up of products will strengthen the company’s market hold in treatments for HAE.  Shire already has two drugs on the market to treat HAE attacks, as well as a preventative drug called Cinryze, which the FDA approved for pediatric use earlier this year.

Andreas Busch, Ph.D., Executive Vice President, Head of Research and Development at Shire said: “With the approval of TAKHZYRO, HAE patients have an innovative treatment that works differently than current options to help prevent attacks. . . This approval reinforces our ongoing commitment to developing novel therapies that have a meaningful impact on patients. Looking to the future, we continue to work towards our goal of a world in which those living with HAE can aim for zero attacks.”

Shire llc is based in Lexington, MA, and also has a large presence in Cambridge, MA, making it one of the largest pharmaceutical companies in Massachusetts by employee headcount.  Shire obtained Takhzyro as a result of its 2015 acquisition of Burlington-based Dyax Corp.

The New England Council congratulates Shire on this key approval and commends the company for its long-standing commitment to developing treatments to treat rare diseases and save lives.  Read more in Shire’s press release or in the Boston Business Journal.

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