FDA grants breakthrough therapy designation to Ayvakit for systemic mastocytosis
The FDA granted breakthrough therapy designation to avapritinib for treatment of moderate to severe indolent systemic mastocytosis, according to the agent’s manufacturer.
Avapritinib (Ayvakit, Blueprint Medicines) is a selective KIT and platelet-derived growth factor receptor alpha (PDGFRA) inhibitor.
A Blueprint Medicines-issued press release indicated company officials intend to present registrational data from the PATHFINDER trial of avapritinib in the first half of this year. The open-label, single-arm phase 2 trial evaluated the efficacy and safety of avapritinib for patients with advanced systemic mastocytosis, including those with aggressive systemic mastocytosis, systemic mastocytosis associated with hematologic neoplasm, and mast cell leukemia.
Company officials hope to obtain FDA approval and launch avapritinib for this indication in the United States in the second half of this year.
The FDA previously approved avapritinib for treatment of patients with unresectable or metastatic gastrointestinal stromal tumors with PDGFRA exon 18 mutations.