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A drug made by Blueprint Medicines markedly improved symptoms of a rare immune system disorder called non-advanced systemic mastocytosis in a clinical trial that is being closely watched by investors, the company said Wednesday.

Blueprint’s stock has risen 28% to $70 this month in anticipation of the results, and the data, though inarguably positive, will lead to more discussion about the drug’s safety and efficacy as well as Blueprint’s pricing strategy for it. Called Ayvakit, it is already approved for two other indications and has a monthly wholesale cost of $35,213.


Fouad Namouni, Blueprint’s head of research and development, said that the result was “an important day” — both for the approximately 30,000 patients who have the disease, most in the non-advanced form treated in this trial, and for researchers at Blueprint who have been working on the drug almost since the company’s 2008 inception.

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