Data from FDA Approval Summary: Ripretinib for Advanced Gastrointestinal Stromal Tumor

• DOI: 10.1158/1078-0432.c.6532875.v1
• Publication Date: 2023-04-01T03:16:04Z
• Authors: Vaibhav Kumar; Leslie Doros; Margaret Thompson; Sirisha L. Mushti; Rosane Charlab; Elizabeth I. Speh...; Hong Zhao; Matthew D. Thompson; Shenghui Tang; Richard Pazdur; Steven J. Lemery; Marc R. Theoret; Lola A. Fashoyin-Aje
• Keywords: Progression-free survival

<div>Abstract<p>On May 15, 2020, the FDA approved ripretinib for adult patients with advanced gastrointestinal stromal tumor who have received prior treatment three or more kinase inhibitors, including imatinib. The approval was based on results from INVICTUS (NCT03353753), an international, multi-center, double-blind, placebo-controlled trial. Patients were randomly allocated (2:1) to receive either 150 mg once daily (<i>n</i> = 85) matching placebo 44). trial demonstrated a statistically significant improvement in progression-free survival (PFS) as assessed by modified RECIST v1.1 blinded independent central review randomized ripretinib, median PFS of 6.3 months [95% confidence interval (CI): 4.6–6.9] compared 1.0 month (95% CI: 0.9–1.7) [HR: 0.15 0.09–0.25); <i>P</i> < 0.0001, stratified log-rank test]. There no difference objective response rate arm, 9% 4.2–18) 0% [(95% 0–8); 0.0504, Fisher exact overall (OS) arm 15.1 12.3–15.1) 6.6 4.1–11.6) arm. A formal statistical comparison OS not made due prespecified hierarchical analysis plan. most common (≥20%) adverse events order decreasing frequency, alopecia, fatigue, nausea, abdominal pain, constipation, myalgia, diarrhea, decreased appetite, palmar-plantar erythrodysesthesia, and vomiting. Other important risks include new primary cutaneous malignancies, hypertension, cardiac dysfunction.</p></div>