Blueprint Medicines Receives FDA Approval for AYVAKIT® (avapritinib) as the First and Only Treatment for Indolent Systemic Mastocytosis (ISM)

Blueprint Medicines Corporation announced that the U.S. Food and Drug Administration (FDA) has approved AYVAKIT® (avapritinib) for the treatment of adults with Indolent Systemic Mastocytosis (ISM). All U.S. patients with ISM now have access to the first and only approved medicine designed to treat their disease.

Systemic Mastocytosis (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.

“After decades of caring for people with Indolent Systemic Mastocytosis, I have seen firsthand its profound impact on patients’ underlying mast cell burden, symptoms, physical and mental health, and ability to work and participate in daily activities,” said Cem Akin, M.D., Ph.D., Professor of Medicine at the University of Michigan,1 and an investigator on the PIONEER trial.1 “Despite the use of multiple supportive care treatments, a considerable number of patients with Indolent Systemic Mastocytosis continue to experience a substantial disease burden. AYVAKIT advances the treatment of Indolent Systemic Mastocytosis by targeting KIT D816V, the primary underlying cause of the disease, and establishes a new standard of care for a broad population of patients with this disorder. AYVAKIT delivered statistically significant and consistent clinical improvements in the PIONEER trial, and based on these practice-changing data, I feel a tremendous sense of hope for the future for all those affected by the disease.”

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 (AYVAKIT) or placebo plus best supportive care (placebo). AYVAKIT demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.

Blueprint Medicines is committed to patient access and affordability for its approved medicines through YourBlueprint®, which is designed to provide dedicated, personalized assistance for eligible patients. Commercially insured patients who qualify for the company’s co-pay assistance program may pay as little as $0 in out-of-pocket costs for AYVAKIT. YourBlueprint offers additional programs intended to facilitate timely access to therapy, including a patient assistance program for eligible patients who have inadequate insurance coverage. For more information, visit or call 1-888-BLUPRNT (1-888-258-7768), Monday to Friday, 8:00 a.m. to 8:00 p.m. ET. Healthcare providers who prescribe AYVAKIT can fill out an enrollment form at to help patients access Blueprint Medicines’ support programs.