Blueprint offers some hope for systemic mastocytosis

For patients with systemic mastocytosis, a rare disorder that causes unpredictable allergic reactions, simply sniffing a perfume-scented magazine page could trigger anaphylactic shock.

Blueprint Medicines is developing a new drug that targets the genetic mutation causing SM. In a small clinical trial, the treatment significantly reduced patients’ symptoms and improved the underlying effects of the disease, STAT’s Damian Garde writes. The drug, avapritinib, works by blocking a pair of enzymes that dictate how immune cells behave. Blueprint is now planning out a larger study for the drug.

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