Pharma Focus Asia

Blueprint Medicines Announces U.S. FDA Approval of AYVAKIT® (avapritinib)

Monday, May 22, 2023

Blueprint Medicines announced that the U.S. Food and Drug Administration (FDA) has approved AYVAKIT® (avapritinib) for the treatment of adults with indolent systemic mastocytosis (ISM). 

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.

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 has delivered statistically significant and consistent clinical improvements in the PIONEER trial, and based on these practise-changing data, it gives a tremendous sense of hope for the future for all those affected by the disease.

Systemic mastocytosis (SM) is a rare haematologic disorder that can lead to a range of debilitating symptoms across multiple organ systems and a significant impact on patients' quality of life. All U.S. patients with ISM now have access to the first and only approved medicine designed to treat the disease.

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