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EMA's CHMP Advises Conditional Marketing Authorization for Blueprint's Ayvakyt in PDGFRA-Mutant GIST

NEW YORK – The European Medicines Agency's Committee for Medicinal Products for Human Use has recommended conditional marketing authorization for avapritinib (Blueprint Medicines' Ayvakyt) for adults with unresectable or metastatic gastrointestinal stromal tumors with a PDGFRA D842V mutation.

The European Commission will now review CHMP's opinion and decide on marketing authorization by the end of September. If approved by the EC, avapritinib will be the first drug available for GIST patients in this molecularly defined indication in the European Union.

CHMP decided to grant conditional marketing authorization to avapritinib based on the safety and efficacy results of the Phase I NAVIGATOR trial and additional safety results from the Phase III VOYAGER trials. In The Lancet Oncology, researchers recently reported that in the NAVIGATOR trial 88 percent of GIST patients with PDGFRA D842V mutations saw their tumors shrink after receiving avapritinib, and the drug had a manageable toxicity profile. 

Approximately 6 percent of GIST patients have mutations in PDGFRA exon 18 mutations, and they don't respond to standard therapies for GIST. The US Food and Drug Administration approved avapritinib in January for unresectable or metastatic GIST with PDGFRA exon 18 mutations, including D842V mutations, which are the most common.