Blueprint Medicines Corporation recently presented updated data showing sustained clinical efficacy and tolerance of Ayvakit (avapritinib) treatment for systemic mastocytosis (SM).
The data was reported across two conferences: the 2025 European Hematology Association (EHA2025) Hybrid Congress held Jun. 12 to 15 in Milan, Italy, and the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2025 held Jun. 13 to 16 in Glasgow, United Kingdom.
Ayvakit is a prescription medication taken once daily by mouth and is approved by the U.S. Food & Drug Administration to treat SM and gastrointestinal stromal tumor.
The presentation included findings from a recently published study in the Journal of Allergy and Clinical Immunology: In Practice, which demonstrated sustained clinical benefits across quality-of-life measures in people taking Ayvakit for indolent SM (ISM). The participants were part of a previous trial using Ayvakit which showed robust improvements in skin, gastrointestinal and neurocognitive symptoms associated with the disorder through 144 weeks.
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Two other trials demonstrated improved overall survival in people living with advanced SM when indirectly compared to real-world data for midostaurin, another oral medication used for the treatment of aggressive SM.
In a press release, Blueprint Medicines Corporation emphasized that Ayvakit continues to be well tolerated in long-term use, with no new safety signals emerging.
“Ayvakit has shown transformative clinical outcomes for patients across the spectrum of SM, including sustained disease control in ISM and prolonged survival in advanced SM,” Dr. Becker Hewes, chief medical officer at Blueprint Medicines, said in a statement. “These compelling results have translated into real-world practice, with clinicians expanding their view of who is an appropriate candidate for disease-modifying therapy after positive Ayvakit experiences, and treatment durations trending toward multiple years.”
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