Blueprint Medicines Receives Positive CHMP Opinion for Avapritinib for the Treatment of Adults with Unresectable or Metastatic PDGFRA D842V Mutant Gastrointestinal Stromal Tumors
The CHMP opinion will now be reviewed by the
"Avapritinib has shown unprecedented clinical activity in patients with PDGFRA D842V mutant GIST, who have traditionally had poor prognoses," said
The CHMP based its opinion on efficacy results from the Phase 1 NAVIGATOR trial as well as combined safety results from the NAVIGATOR and Phase 3 VOYAGER trials. Treatment with avapritinib showed deep and durable clinical responses and was well-tolerated in patients with PDGFRA D842V mutant GIST. Data in this patient population were published in The Lancet Oncology on
Avapritinib is a kinase inhibitor approved by the
Avapritinib is not approved for the treatment of any other indication in the
GIST is a sarcoma, or tumor of bone or connective tissue, of the GI tract. Tumors arise from cells in the wall of the GI tract and occur most often in the stomach or small intestine. Most patients are diagnosed between the ages of 50 to 80, and diagnosis is typically triggered by GI bleeding, incidental findings during surgery or imaging and, in rare cases, tumor rupture or GI obstruction.
About 5 to 6 percent of primary GIST cases are caused by a PDGFRA D842V mutation, the most common PDGFRA exon 18 mutation. Prior to the FDA approval of AYVAKIT, there were no highly effective treatments for PDGFRA D842V mutant GIST in the
Blueprint Medicines is a precision therapy company striving to improve human health. With a focus on genomically defined cancers, rare diseases and cancer immunotherapy, we are developing transformational medicines rooted in our leading expertise in protein kinases, which are proven drivers of disease. Our uniquely targeted, scalable approach empowers the rapid design and development of new treatments and increases the likelihood of clinical success. We have one FDA-approved precision therapy and are currently advancing multiple investigational medicines in clinical development, along with a number of research programs. For more information, visit www.BlueprintMedicines.com and follow us on Twitter (@BlueprintMeds) and LinkedIn.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the expectations and timing for a decision from the European Commission on the marketing authorization application for avapritinib for the treatment of adults with PDGFRA D842V mutant GIST; the potential benefits of avapritinib in treating patients; timing and plans for commercialization of avapritinib in the
1 Cassier PA, Fumagalli E, Rutkowski P., et al. Outcome of patients with platelet-derived growth factor receptor alpha-mutated gastrointestinal stromal tumors in the tyrosine kinase inhibitor era.
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