Shire Wins Approval for Rare-Disease Drug Developed At Boston-Based Unit
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players in the inflammatory pathway, including drugs made by Cambridge, MA-based Dyax (NASDAQ: DYAX) and Exton, PA-based ViroPharma (NASDAQ: VPHM). The ViroPharma drug can be self-dosed, but is designed to prevent acute attacks, not treat them. And the Dyax drug, which is approved for acute attacks, can’t be self-administered. “Ours is portable, you can store it at room temperature,” Cole says. “Patients can use it and keep going with their day. It gives them a lot of freedom to manage their disease.” [An earlier version of the paragraph mistakenly said the ViroPharma product couldn't be self-administered. We regret the error. Further details have been added to describe the differences between the drugs and how they're used.]
Shire may be able to tout a safety advantage, as well. Dyax’s drug, which was approved in December 2009, carries a prominent “black box” warning about potentially life-threatening allergic reactions. Shire’s drug was cleared without a black box warning.
Shire hasn’t estimated the market size for icatibant in dollars. But Dyax’s sales do provide a preview of the opportunity. In 2010, Dyax sold $8.8 million worth of its drug, and it is well on track to double that this year. In the most recent quarter, Dyax reported $5.2 million in sales and a patient base that grew 50 percent over the previous quarter.
The approval of icatibant is the latest feather in Shire’s rare-disease cap. The company, which is headquartered in Dublin, Ireland, made its first foray into orphan diseases back in 2005, when it bought Cambridge-based Transkaryotic Therapies (TKT) for $1.6 billion. That unit, now Human Genetic Therapies, is responsible for some of Shire’s biggest hits, including velaglucerase alfa (Vpriv) for Gaucher disease, and agalsidase alfa (Replagal) for Fabry disease. Shire CEO Angus Russell told Xconomy in May that targeting small patient populations could be lucrative for companies that go to market with clear advantages over the standard of care or the competition.
Cole is confident the company’s long history in rare diseases will serve it well in the competitive angioedema market. He says that Shire will provide a wide range of services for patients, including case managers they can call to talk about issues such as insurance reimbursement and managing their disease. “Part of our emphasis on rare diseases is providing those services,” he says. “We think we do that well.”