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.
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The US FDA gave the drug a broad label in adults with indolent SM, without restrictions for disease severity or prior treatment, and Blueprint sees a potential $1.5bn market opportunity.
The Phase III PIONEER study in indolent systemic mastocytosis met the primary endpoint, but the magnitude of benefit came up short of analysts’ expectations.
The company anticipates potential sales in excess of $1bn for the late-onset Pompe disease drug by the end of the decade.