Blueprint Medicines Gets FDA Priority Review for Ayvakit in Indolent Systemic Mastocytosis

By Colin Kellaher

Blueprint Medicines Corp. on Monday said the U.S. Food and Drug Administration granted priority review to its application seeking expanded approval of Ayvakit for adults with indolent systemic mastocytosis, a rare blood disorder.

The Cambridge, Mass., company said the application is based on its registrational "Pioneer" study, in which Ayvakit achieved its primary and all key secondary endpoints, with highly meaningful reductions in patient-reported symptoms and all measures of mast cell burden studied.

The FDA granted priority review to medicines that have the potential to provide significant improvements in the treatment of a serious disease, and the designation shortens the review period. Blueprint said the agency has set a target action date of May 22 for its application.

The FDA in 2021 approved Ayvakit for adults with advanced systemic mastocytosis, including patients with aggressive systemic mastocytosis, systemic mastocytosis with an associated hematological neoplasm, and mast cell leukemia.

Blueprint reported net product revenue of $28.6 million from sales of the drug in the third quarter of 2022.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

01-23-23 0836ET