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Blueprint’s Ayvakit Mastocytosis Results Raise Commercial Uncertainty

 
• By Alaric DeArment

The Phase III PIONEER study in indolent systemic mastocytosis met the primary endpoint, but the magnitude of benefit came up short of analysts’ expectations.

Blueprint Medicines announced Phase III data for Ayvakit in indolent systemic mastocytosis • Source: Shutterstock

Blueprint Medicines Corporation’s Phase III PIONEER trial data for Ayvakit (avapritinib) in indolent systemic mastocytosis (ISM) will likely prove sufficient for US Food and Drug Administration approval, but it has raised questions about how much it will drive commercial adoption, as the magnitude of benefit shown in the trial came in below what investors had hoped for.

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