Posted by Michael Wonder on 09 Jan 2020
FDA approves the first targeted therapy to treat a rare mutation in patients with gastro-intestinal stromal tumours
9 January 2020 - Today, the U.S. FDA approved Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumour (GIST) harboring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation.
This approval includes GIST that harbors a PDGFRA D842V mutation, which is the most common exon 18 mutation. Ayvakit is a kinase inhibitor, meaning it blocks a type of enzyme called a kinase and helps keeps the cancer cells from growing.
The FDA approved Ayvakit based on the results of a clinical trial involving 43 patients with GIST harbouring a PDGFRA exon 18 mutation, including 38 patients with PDGFRA D842V mutation.
