Blueprint Medicines secures FDA approval for Ayvakit

Blueprint Medicines has secured the US Food and Drug Administration (FDA) approval for Ayvakit (avapritinib) for treating unresectable or metastatic gastrointestinal stromal tumour among adults (GIST).

Ayvakit, a kinase inhibitor, is the first precision therapy to be approved to treat GIST harbouring a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation. The approval also extends to GIST with PDGFRA D842V mutation.

GIST is a genomically driven sarcoma of the gastrointestinal (GI) tract that usually develops in the stomach or small intestine. Around 6% of GIST patients exhibit PDGFRA exon 18 mutations.

The FDA approved Ayvakit for GIST based on the results of Phase I NAVIGATOR clinical trial. The trial included 43 patients with GIST harbouring a PDGFRA exon 18 mutation and 38 patients with PDGFRA D842V mutation.

Ayvakit demonstrated an overall response rate (ORR) of 84% in patients with PDGFRA exon 18 mutant GIST.

Common side effects among patients included oedema, nausea, fatigue / asthenia, vomiting, decreased appetite and diarrhoea.

NAVIGATOR trial investigator Michael Heinrich said: "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.

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

Blueprint Medicines plans to distribute Ayvakit in the US within a week.

Blueprint Medicines CEO Jeff Albers said: "The full approval of AYVAKIT based on robust data from our Phase I NAVIGATOR clinical trial is an incredibly exciting milestone for our company and, more importantly, for GIST patients with a PDGFRA exon 18 mutation, who have been waiting for a new treatment option."

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