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Blueprint Medicines wins FDA approval for drug to treat rare blood disorder

Blueprint Medicines CEO Jeff Albers.
Blueprint Medicines CEO Jeff Albers.Suzanne Kreiter/Globe Staff

Blueprint Medicines won approval Wednesday for a precision medicine to treat advanced systemic mastocytosis, a rare blood disorder.

It’s the Cambridge biotech’s fourth approval from the Food and Drug Administration in less than two years. The drug cleared Wednesday, called Ayvakit, had previously been approved for an ultra-rare form of gastrointestinal cancer.

Systemic mastocytosis is a life-threatening disease that results in too many mast cells building up in the body. Symptoms such as anaphylaxis, fatigue, and bone pain can be debilitating, limiting a patient’s ability to perform daily tasks.

The drug was approved to treat the three subtypes of the blood disorder, which include mast cell leukemia. Blueprint’s medicine, taken orally, is the first therapy designed to target a mutation that 95 percent of patients with the disease have.


Blueprint’s stock closed up 1.5 percent at $85.56. The biotech was launched in 2011 with funding from Boston-based Third Rock Ventures and became a publicly traded company in 2015.

Anissa Gardizy can be reached at anissa.gardizy@globe.com. Follow her on Twitter @anissagardizy8.