FDA Approves AYVAKIT® (avapritinib) as the First and Only Treatment for Indolent Systemic Mastocytosis

Tuesday, May 23, 2023

Blueprint Medicines Corporation today 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.

"Today's approval of AYVAKIT reflects more than a decade of collaboration and leadership in the field of systemic mastocytosis, during which our team at Blueprint Medicines has worked closely with clinicians, patients and their families, and patient advocacy groups to advance innovative science and develop a transformational treatment for people living with the disease," said Becker Hewes, M.D., Chief Medical Officer at Blueprint Medicines. "AYVAKIT is the first and only medicine approved by the FDA to treat indolent systemic mastocytosis, marking a shift in the treatment paradigm from supportive care to disease modifying therapy. In addition, AYVAKIT is the only treatment approved across the spectrum of indolent and advanced systemic mastocytosis. With a broad indication for indolent systemic mastocytosis and a strong label, we are now engaging healthcare providers to redefine what it means for their patients to be well-controlled as well as activating the patient community to seek out optimal care and treatment."

"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.

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 (≥10 percent) were eye edema, dizziness, peripheral edema and flushing. Serious adverse reactions and discontinuations due to adverse reactions occurred in less than 1 percent of patients.

Detailed results from the PIONEER trial, including open-label extension study data showing the clinical benefits of AYVAKIT through 48 weeks of treatment, were presented in February 2023 at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting.

"People with indolent systemic mastocytosis are significantly impacted by their disease symptoms, and many individuals self-isolate at home to protect against unpredictable external triggers. Ultimately, the disease can disrupt an individual's quality of life, career and ability to interact with family and friends, and those affected often face frequent healthcare visits and significant polypharmacy burden," said Judith Kain Emmel, Board Chair of The Mast Cell Disease Society. "Since we began nearly 30 years ago, our goals as an organization have been to educate, advocate and collaborate for the advancement of knowledge and research in mast cell disorders like systemic mastocytosis. Today's approval is a historic moment for the SM community and offers new hope for patients and their families. We are honored to have contributed to this milestone by facilitating SM community insights on the design of the PIONEER trial and supporting the development of the validated symptom assessment tool used in the study."

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.



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