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FDA Approves Avapritinib for PDGFRA Exon 18+ GIST

Gina Columbus @ginacolumbusonc
Published: Thursday, Jan 09, 2020

In the PDGFRA exon 18–mutant cohort, 67.4% of patients were white, 88.4% of patients harbored a PDGFRA D842V mutation, and the median number of prior therapies was 1. A total of 97.7% of patients had metastatic disease, 46.5% of patients had a target lesion ≤5 cm, and 86.0% of patients had prior surgical resection. Patients had an ECOG performance status of 0 (32.6%), 1 (60.5%), or 2 (7.0%).

exon 18 mutation, who have been waiting for a new treatment option," Jeff Albers, chief executive officer at Blueprint Medicines, stated in the press release. "Ayvakit is the first of what we hope will be many approved medicines enabled by our research platform. Now, as we begin to deliver Ayvakit to patients and their healthcare providers, we aim to fortify our leadership in the field of precision medicine and build a foundation for our broader portfolio by pairing our strong research and development capabilities with an equally talented commercial organization focused on addressing patient needs, accelerating diagnostic testing and enabling access."


  1. Blueprint Medicines announces FDA approval of Ayvakit (avapritinib) for the treatment of adults with unresectable or metastatic PDGFRA exon 18 mutant gastrointestinal stromal tumor [news release]. Blueprint Medicines Corporation. Published January 9, 2020. Accessed January 9, 2020. 
  2. Blueprint Medicines Announces FDA Acceptance of New Drug Application for Avapritinib for the Treatment of PDGFRA Exon 18 Mutant GIST and Fourth-Line GIST. Blueprint Medicines Corporation. Published August 7, 2019. Accessed August 7, 2019.
  3. Blueprint Medicines Announces FDA Intent to Split Avapritinib New Drug Application into Separate Submissions for PDGFRA Exon 18 Mutant GIST and Fourth-Line GIST. Blueprint Medicines Corporation. Published October 28, 2019. Accessed October 28, 2019.
  4. Heinrich M, Jones RL, von Mehren M, et al. Clinical activity of avapritinib in ≥4th line (4L+) and PDGFRA exon 18 gastrointestinal stromal tumors (GIST). J Clin Oncol. 2019;37 (suppl; abstr 11022).

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