Blueprint Medicines(US:BPMC) Prnewswire·2024-05-30 22:01Core Insights - Blueprint Medicines Corporation is advancing the understanding and treatment of systemic mastocytosis (SM) through multiple upcoming datasets presented at key conferences, emphasizing the real-world burden of the disease and the clinical outcomes of AYVAKIT/AYVAKYT (avapritinib) [1][2][5] Group 1: Clinical Efficacy and Safety of AYVAKIT - AYVAKIT demonstrated durable efficacy and a favorable safety profile in patients with indolent systemic mastocytosis (ISM) over a median follow-up of more than two years, with safety data consistent for approximately 10% of patients who escalated to a 50 mg daily dose [1][2] - The median overall survival (OS) for AYVAKIT in advanced SM has not been reached, with a historical median OS ranging from less than six months to about 3.5 years, and an overall response rate of 73% [3][4] - AYVAKIT showed sustained improvements in total symptom scores and quality of life, with the most common treatment-related adverse event being edema, primarily Grade 1, and a low discontinuation rate of 3% due to treatment-related adverse events [2][4] Group 2: Patient Perspectives and Disease Burden - The PRISM Survey highlighted the significant burden of systemic mastocytosis on patients' quality of life, with many reporting debilitating symptoms and impaired physical and mental health functioning [5][6] - Healthcare providers acknowledged the impact of SM, with 65% indicating it affected patients' lives "quite a bit" or "a great deal," and 58% noting that patients lost employment opportunities due to the disease [6] Group 3: Upcoming Presentations and Research - Data presentations will be made available at the European Academy of Allergy and Clinical Immunology (EAACI) Congress 2024 and the European Hematology Association 2024 (EHA24) Hybrid Congress, covering various aspects of AYVAKIT's safety, efficacy, and the burden of SM [7][8]