Blueprint Presents Positive Ayvakit Data, But Commercial Debate Could Continue

Detailed data in indolent systemic mastocytosis show improvement across primary and key secondary endpoints, but analysts pointed to lower improvement on symptoms outside of those affecting the skin.  

Blueprint unveiled detailed data for the Phase III PIONEER study of Ayvakit in indolent systemic mastocytosis • Source: Shutterstock

Blueprint Medicines Corporation’s presentation of full data from the Phase III PIONEER study of Ayvakit (avapritinib) in indolent systemic mastocytosis (ISM) bolsters the drug’s chances of winning approval while allaying some – but not all – of the concerns about its commercial potential in that disease amid a debate that arose when the company announced topline results from the study in 2022.

The biotech presented the data on 26 February at the American Academy of Allergy, Asthma & Immunology (AAAI) meeting. They are part of a supplemental new drug application undergoing priority...

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