BPMC - Blueprint to seek label expansion for systemic mastocytosis therapy after trial win

• Blueprint Medicines ( NASDAQ: BPMC ), the maker of systemic mastocytosis therapy Ayvakit, said Tuesday that it would file a supplemental new drug application to the FDA this year after the treatment reached main goals in a registration trial for patients with the non-advanced form of the disease.
• Systemic mastocytosis is a genetically driven rare disease leading to symptoms such as anaphylaxis, maculopapular rash, pruritis, diarrhea, brain fog, fatigue, and bone pain.
• Ayvakit, also known as avapritinib, is a kinase inhibitor approved in the U.S. for diseases including advanced SM.
• According to topline data, Part 2 of the PIONEER clinical trial met the primary and all key secondary endpoints, indicating clinically meaningful and highly significant improvements for measures such as patient-reported symptoms and objective measures of mast cell burden.
• The company plans to disclose detailed data at an upcoming medical meeting.
• In addition to an sNDA filing expected in 4Q 2022 in the U.S., BPMC plans to submit a type II variation marketing authorization application to the European drug regulator in 2023.

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