Blueprint Medicines receives FDA OK for Ayvakit

Blueprint Medicines has obtained the Food and Drug Administration’s approval for Ayvakit (avapritinib) for the treatment of adult patients with advanced systemic mastocytosis, including aggressive SM, SM with an associated hematological neoplasm and mast cell leukemia.

"Today's approval of Ayvakit for advanced systemic mastocytosis – the fourth FDA approval across our portfolio in 18 months — culminates nearly a decade of hard work from our scientists in the laboratory and clinical team conducting trials to our commercial organization who will now bring Ayvakit to patients," said Jeff Albers, Blueprint Medicines CEO.

"As shown in two clinical trials, Ayvakit provides remarkable clinical efficacy to patients with advanced systemic mastocytosis, and this approval solidifies the therapy's strong value proposition in this population," Albers said. With a deep commitment to driving continued research innovation in collaboration with the mast cell disease community, we are now building on this progress with the goal of bringing the benefits of precision therapy to a broader range of patients through our ongoing and planned clinical trials for non-advanced systemic mastocytosis."

For the first time, advanced SM patients can now receive a targeted therapy designed to potently and selectively inhibit D816V mutant KIT, the central driver of the disease, the company said. 

"Advanced systemic mastocytosis is a debilitating disease characterized by extensive damage in multiple organ systems due to mast cell infiltration, and new treatment options are urgently needed to address these life-threatening complications," said Daniel DeAngelo, chief of the division of leukemia at Dana-Farber Cancer Institute.

"Avapritinib will clearly establish a new standard of care for patients with advanced systemic mastocytosis. The FDA approval was based on data showing robust and durable responses, including complete remissions, and a favorable safety profile," DeAngelo said. "For advanced SM patients, the approval of avapritinib shifts the treatment paradigm toward precision therapy that targets the primary driver of mastocytosis."