Blueprint Medicines Presents Strong Long-Term Data for AYVAKIT in Systemic Mastocytosis

Blueprint Medicines Corporation (NASDAQ:BPMC) is one of the best biotech stocks to invest in now. On June 12, Blueprint Medicines announced new data reinforcing the sustained clinical efficacy and well-tolerated safety profile of long-term AYVAKIT/AYVAKYT (avapritinib) treatment. These findings were presented at the 2025 European Hematology Association Hybrid Congress, which was held from June 12-15, in Milan, Italy, and the European Academy of Allergy and Clinical ImmunologyCongress 2025, held from June 13-16, in Glasgow, UK.

The presentations highlighted over a decade of collaboration to transform systemic mastocytosis/SM treatment. The data were derived from large patient populations in the PIONEER, PATHFINDER, and EXPLORER trials and featured follow-up periods of up to 5 years in Indolent Systemic Mastocytosis/ISM and up to 6.5 years in advanced SM. The extensive follow-up demonstrates the favorable long-term benefits of AYVAKIT and the significant datasets amassed by Blueprint Medicines.

Blueprint Medicines Presents Strong Long-Term Data for AYVAKIT in Systemic Mastocytosis

A doctor examining a patient’s samples in a modern hospital setting.

SM is a rare disease primarily driven by the KIT D816V mutation in ~95% of cases. It leads to uncontrolled mast cell proliferation and activation, which results in chronic, severe, and unpredictable symptoms across multiple organ systems. AYVAKIT (avapritinib) is the first and only medicine approved by the US FDA to treat the root cause of SM. It received FDA approval for advanced SM in June 2021 and for ISM in May 2023.

Blueprint Medicines Corporation (NASDAQ:BPMC) is a precision therapy company that develops medicines for genomically defined cancers and blood disorders in the US and internationally.

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Disclosure: None. This article is originally published at Insider Monkey.