Blueprint Medicines Announces European Commission Approval of AYVAKYT® (avapritinib) for the Treatment of Adults with Unresectable or Metastatic PDGFRA D842V Mutant Gastrointestinal Stromal Tumors
GIST is a genomically driven sarcoma of the gastrointestinal (GI) tract, with PDGFRA D842V mutations implicated in a rare subset of patients. A retrospective study of patients with PDGFRA D842V mutant GIST showed that treatment with imatinib, the standard first-line GIST therapy, led to an ORR of 0 percent.2 ESMO guidelines recommend including mutational testing in the GIST diagnostic work-up as standard practice.
"There have historically been no treatments offering hope for patients with PDGFRA D842V mutant GIST. AYVAKYT represents the first major therapeutic breakthrough for patients with GIST harboring this mutation, defining a new standard of care," said
"The introduction of a new targeted therapy, AYVAKYT, is an important milestone for patients with PDGFRA D842V mutant GIST, who have historically had no effective treatment options," said
About AYVAKYT (avapritinib)
AYVAKYT (avapritinib) is a kinase inhibitor approved in the EU for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. AYVAKYT is the first precision therapy approved in
This therapy is not approved for the treatment of any other indication in the U.S. or
Blueprint Medicines has an exclusive collaboration and license agreement with CStone Pharmaceuticals for the development and commercialization of avapritinib in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.
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. Published data have shown poor outcomes in patients with PDGFRA D842V mutant GIST treated with imatinib and other approved therapies, including a median OS of 15 months, a median PFS of 3 months and an ORR of 0 percent.2
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
1 AYVAKYT® (avapritinib) Summary of Product Characteristics (EU).
2 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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