US-based precision therapy provider Blueprint Medicines has received the US Food and Drug Administration (FDA) approval for AYVAKIT (avapritinib) to treat a type of gastrointestinal cancer.

The FDA approval indicates AYVAKIT for metastatic gastrointestinal stromal tumour (GIST) with a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, including PDGFRA D842V mutations.

Blueprint Medicines chief executive officer Jeff Albers said: “The full approval of AYVAKIT based on robust data from our Phase 1 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.

“AYVAKIT is the first of what we hope will be many approved medicines enabled by our research platform. Now, as we begin to deliver AYVAKIT to patients and their healthcare providers, we aim to fortify our leadership in the field of precision medicine and build a foundation for our broader portfolio.”

AYVAKIT showed 84% overall response rate in gastrointestinal cancer patients

Blueprint said that its AYVAKIT marks the first precision therapy to be approved by the FDA to treat a genomically defined population of patients with GIST, a rare, genomically driven sarcoma of the gastrointestinal (GI) tract.

The regulatory approval of AYVAKIT is supported by the results from the Phase 1 NAVIGATOR clinical trial, along with safety results from various clinical trials for avapritinib.

In addition, AYVAKIT demonstrated 84% overall response rate (ORR) in the clinical study of gastrointestinal cancer patients with PDGFRA exon 18 mutant GIST.

The most common adverse reactions of the drug include oedema, nausea, fatigue or asthenia, cognitive impairment, vomiting, decreased appetite, diarrhoea, hair colour changes, increased lacrimation, abdominal pain, constipation, rash and dizziness.

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.

“Building on our growing understanding of the molecular basis of GIST, this milestone ushers in a new era of precision medicine in this disease.

“The FDA approval represents a call to action to conduct mutational testing in all patients with GIST before initiating kinase inhibitor therapy, as recommended by clinical guidelines, so appropriate patients may realize the benefits of this promising new medicine.”