FDA approves first precision therapy for GIST

Blueprint Medicines has gained the green light in the US for Ayvakit for treating adults with unresectable or metastatic gastrointestinal stromal tumour (GIST) harbouring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

Ayvakit (avapritinib) is now the first precision therapy approved to treat a genomically defined population of patients with GIST, a rare, genomically driven sarcoma of the gastrointestinal tract.

The approval was based on efficacy results from the Phase 1 NAVIGATOR clinical trial, as well as combined safety results from multiple clinical trials for avapritinib.

In patients with PDGFRA exon 18 mutant GIST, Ayvakit had an overall response rate (ORR) of 84%, and a median duration of response (DOR) was not reached.

“Today’s approval of AYVAKIT brings forward a new standard of care for patients with PDGFRA exon 18 mutant GIST, a genomically defined population that previously had very limited treatment options,” said Michael Heinrich, Professor of Medicine at Oregon Health & Science University and an investigator on the NAVIGATOR trial.

“For the first time, we can offer these patients a highly effective treatment that targets the underlying genetic cause of their disease.”

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