-- Real-world study of AYVAKIT highlights improvement across ISM symptoms --

-- 12 data presentations at 2026 AAAAI Annual Meeting reflect company's leadership in elevating SM care --

Blueprint Medicines, a Sanofi company,  announced AYVAKIT^® (avapritinib) data showing clinically meaningful outcomes and a well-tolerated long-term safety profile in patients with indolent systemic mastocytosis (ISM). Key presentations include four-year PIONEER clinical study results highlighting sustained symptom control and quality of life improvement in AYVAKIT-treated patients, and real-world data showing meaningful symptom benefit in patients receiving the therapy in a community practice setting.

In addition, a study conducted with the Advanced Practitioner Society for Hematology and Oncology (APSHO) and The Mast Cell Disease Society (TMS) reinforces the severe symptoms and impaired quality of life experienced by patients with ISM. In total, 12 data presentations are being reported at the 2026 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting, February 27 – March 2 in Philadelphia.

"Patients with indolent systemic mastocytosis and healthcare providers have expressed the need for a therapy that meaningfully improves quality of life through durable symptom benefit and a safety profile enabling long-term treatment, and AYVAKIT is delivering this significant impact to a wide range of people living with the disease," said Mik Rinne, M.D., Ph.D., Head of Development at Blueprint Medicines.

"Across clinical and real-world settings, AYVAKIT has shown robust efficacy and a well-tolerated safety profile, helping patients realize the sustained benefit of KIT D816V-targeted therapy. In addition, emerging evidence continues to underscore the substantial burden of SM, highlighting the urgency to treat the underlying cause of the disease."