Blueprint Medicines Receives FDA Authorization to Advance Novel Drug Candidate into Clinical Trial for Systemic Mastocytosis
"BLU-285 has the potential to be a highly targeted therapy for patients with severe forms of SM who lack effective treatment options," said
BLU-285 is a potent and highly selective inhibitor of the KIT D816V mutant, the primary driver of disease in more than 94 percent of SM patients. SM is characterized by the abnormal accumulation of mast cells. In patients with advanced forms of the disease, mast cells accumulate in organs, such as the bone marrow, intestines, spleen and liver, compromising organ function. There are no approved targeted therapies for SM patients with KIT D816V-driven disease.
In this Phase 1 trial,
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