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Blueprint Medicines announced that NEJM Evidence, a journal from The New England Journal of Medicine Group, has published detailed results from the PIONEER trial of AYVAKIT in patients with indolent systemic mastocytosis. Data reported in the publication show that AYVAKIT achieved statistically significant and clinically meaningful benefits across efficacy measures including disease symptoms and pathological mast cell burden, which continued to improve over time through 48 weeks. In addition, AYVAKIT demonstrated a favorable safety profile relative to placebo. On May 22, 2023, AYVAKIT became the first and only medicine approved by the U.S. Food and Drug Administration for the treatment of adults with ISM. AYVAKIT was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. ISM represents the vast majority of cases of systemic mastocytosis, a rare hematologic disorder that can lead to debilitating symptoms across multiple organ symptoms and a profound impact on patients' quality of life. PIONEER is a randomized, double-blind, placebo-controlled trial in which 141 patients received AYVAKIT 25 mg once daily plus best supportive care and 71 patients received placebo plus best supportive care. Disease symptoms were assessed using the ISM Symptom Assessment Form. As previously reported, AYVAKIT met the primary and all key secondary endpoints, which were evaluated from baseline to 24 weeks, and showed deepening clinical efficacy through 48 weeks of treatment. Overall, 96 percent of patients with AYVAKIT completed 24 weeks of treatment and opted to cross over to the open-label extension study. Key published results include: Patients treated with AYVAKIT achieved rapid, durable and statistically significant reductions on all measures of pathological mast cell burden, including serum tryptase, KIT D816V variant allele fraction and bone marrow mast cells. AYVAKIT achieved statistically significant and clinically meaningful benefits in overall symptoms - as measured by the ISM-SAF Total Symptom Score - compared to placebo at 24 weeks, with improvements deepening through 48 weeks. In addition, AYVAKIT showed improvements over placebo on the most severe symptom and across all individual symptoms measured. AYVAKIT showed clinically meaningful improvements in multiple exploratory endpoints of quality of life, including a disease-specific measure - Mastocytosis Quality of Life Questionnaire- and general health status measures such as European Quality of Life Visual Analogue Scale, Patient Global Impression of Severity and 12-Item Short-Form Health Survey. AYVAKIT showed a favorable safety profile, with most adverse events mild to moderate in severity. The serious AE rate was 5.0 percent for AYVAKIT and 11.3 percent for placebo. The most common treatment-emergent AEs for AYVAKIT were flushing, edema, increased blood alkaline phosphatase and insomnia.

Blueprint Medicines announced that the FDA has approved AYVAKIT, or avapritinib, for the treatment of adults with indolent systemic mastocytosis, or SM. All U.S. patients with ISM now have access to the first and only approved medicine designed to treat their disease. Systemic mastocytosis, or SM, is a rare hematologic disorder that can lead to a range of debilitating symptoms across multiple organ systems and a significant impact on patients' quality of life. ISM represents the vast majority of SM cases, and AYVAKIT is now available for adults with ISM at the recommended dose of 25 mg once daily. AYVAKIT was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. AYVAKIT has been FDA approved for the treatment of advanced SM since June 2021. The approval of AYVAKIT in ISM is based on data from the double-blind, placebo-controlled PIONEER trial - the largest study ever conducted for this disease - in which patients received AYVAKIT 25 mg once daily plus best supportive care or placebo plus best supportive care. AYVAKIT demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden. AYVAKIT was well-tolerated with a favorable safety profile compared to placebo, and most adverse reactions were mild to moderate in severity. The most common adverse reactions for AYVAKIT (greater than or equal to10 percent) were eye edema, dizziness, peripheral edema and flushing. Serious adverse reactions and discontinuations due to adverse reactions occurred in less than 1% of patients.

Blueprint Medicines' Ayvakit received approval for indications that are expanded to include indolent systemic mastocytosis, or ISM, according to updated prescribing information posted to the site of the FDA. Reference Link