FDA rejects Blueprint’s NDA for AYVAKIT for treatment of fourth-line GIST

Blueprint Medicines said that the US Food and Drug Administration (FDA) has rejected its new drug application (NDA) for AYVAKIT (avapritinib) for the treatment of fourth-line gastrointestinal stromal tumor (GIST).

The US precision therapy company received a complete response letter (CRL) from the FDA in this connection.

Blueprint Medicines was seeking FDA approval for avapritinib through the NDA for the treatment of unresectable or metastatic fourth-line gastrointestinal stromal tumor (GIST) in adults.

AYVAKIT, which is a kinase inhibitor, has FDA approval for the treatment of unresectable or metastatic GIST harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations, in adult patients. The precision therapy company intends to continue to commercialize AYVAKIT in the US for the approved indication.

FDA rejects Blueprint’s NDA for AYVAKIT for treatment of fourth-line gastrointestinal stromal tumor. Photo courtesy of The U.S. Food and Drug Administration/Wikipedia.org.

Blueprint Medicines will also look to get marketing approval for avapritinib for the treatment of the same patient population in more geographies, including the European Union.

Additionally, the precision therapy company continues to advance the development of avapritinib for the treatment of systemic mastocytosis (SM).

The company said that based on top-line results of the phase 3 VOYAGER trial which were released last month, it will discontinue further development of avapritinib in GIST indications other than PDGFRA exon 18 mutant GIST.

FDA rejects Blueprint’s NDA for AYVAKIT for treatment of fourth-line GIST added by pharmanewsdaily on May 15, 2020
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