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Blueprint Will Not Develop Ayvakit Beyond PDGFRA Exon 18-Mutated Stomach Cancer After Phase III Fail

NEW YORK – After a Phase III trial failed to demonstrate a progression-free survival advantage for Blueprint Medicines' avapritinib (Ayvakit) over regorafenib (Bayer's Stivarga) in advanced stomach cancer patients, the drugmaker has decided it will stop developing the drug beyond its already approved molecularly defined indication.

The US Food and Drug Administration earlier this year approved avapritinib for unresectable or metastatic gastrointestinal stromal tumors harboring a PDGFRA exon 18 mutation. Blueprint was also trying to expand the drug's indication beyond this biomarker subgroup. 

However, based on the results from the VOYAGER trial, Blueprint said in a statement this week that it will "discontinue further development of avapritinib in GIST beyond PDGFRA exon 18 mutant GIST." In the Phase III study comparing avapritinib and regorafenib, the median progression-free survival was 4.2 months versus 5.6 months, respectively. Additionally, 17 percent of avapritinib-treated patients saw their tumors shrink compared to 7 percent receiving regorafenib.

"We hope these data will reveal important insights to improve the scientific understanding of the disease and inform future innovations in GIST, and we are committed to sharing the results at a future medical meeting," Blueprint CEO Jeff Albers said in a statement.

The company said it will continue commercializing avapritinib in the US in the PDGFRA exon 18-mutated GIST population and seek approval for this indication in other countries. The company is anticipating a decision from the European Commission in the third quarter of 2020.