1/18/2024 0 Comments Nasdaq bpmc news![]() AYVAKIT demonstrated significant improvements versus placebo in the primary and all key secondary endpoints, including overall symptoms and measures of mast cell burden.ĪYVAKIT was well-tolerated with a favorable safety profile compared to placebo, and most adverse reactions were mild to moderate in severity. 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 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." 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. 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. "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. 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." In addition, AYVAKIT is the only treatment approved across the spectrum of indolent and advanced systemic mastocytosis. "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. ![]() "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 has been FDA approved for the treatment of advanced SM since June 2021. ![]() AYVAKIT was designed to potently and selectively inhibit KIT D816V, the primary underlying driver of the disease. 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. 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.
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