Blueprint Medicines Corporation announced that the U.S. Food and Dru 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.
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“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.”
SOURCE: PR Newswire