Blueprint Medicines announced that the FDA has issued a complete response letter (CRL) for the new drug application of Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic fourth-line gastrointestinal stromal tumor (GIST). The CRL states that the FDA cannot approve the application.
As previously announced, Blueprint Medicines plans to continue to commercialize Ayvakit (avapritinib) in the United States for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, and seek marketing approval for avapritinib for the treatment of this patient population in additional geographies, including the European Union.
In addition, Blueprint Medicines continues to advance development of avapritinib for the treatment of systemic mastocytosis (SM). Based on top-line results reported in April 2020 for Blueprint Medicines’ Phase III VOYAGER trial, the company previously announced plans to discontinue further development of avapritinib in GIST indications other than PDGFRA exon 18 mutant GIST.