ADVERTISEMENT
FDA Approves Ripretinib for Pretreated Adults With Advanced GIST
On May 15, 2020, the FDA approved ripretinib (Qinlock; Deciphera Pharmaceuticals) for the treatment of adults with advanced gastrointestinal stromal tumor (GIST) who have received therapy with ≥3 or more kinase inhibitors, including imatinib.
This approval was based on efficacy data from the international, multi-center INVICTUS clinical trial, in which 129 patients with GIST who previously received imatinib, sunitinib, and regorafenib were randomized in a 2:1 ratio to receive ripretinib 150 mg or placebo once daily until disease progression or unacceptable toxicity occurred.
Patients randomized to the placebo arm could crossover to the ripretinib arm at the point of disease progression.
The major efficacy end point was progression-free survival (PFS), and other end points included overall response rate (ORR) and overall survival (OS).
Ultimately, patients in the ripretinib arm had a statistically significant improvement in PFS compared with those in the placebo arm (hazard ratio [HR], 0.15; 95% CI, 0.09-0.25; P <.0001); the median PFS was 6.3 months (95% CI, 4.6-6.9) with ripretinib versus 1.0 month (95% CI, 0.9-1.7) with placebo.
Although the ORR was 9% (95% CI, 4.2-18) with ripretinib versus 0% (95% CI, 0-8) with placebo, this difference was not deemed statistically significant.
Furthermore, although the median OS was 15.1 months (95% CI, 12.3-15.1) versus 6.6 months (95% CI, 4.1-11.6), respectively (HR, 0.36; 95% CI, 0.21-0.62), the OS was not evaluated for statistical significance because of the sequential testing procedure for the secondary end points.
The most common (≥20%) adverse events associated with ripretinib use were alopecia, fatigue, nausea, abdominal pain, constipation, myalgia, diarrhea, decreased appetite, palmar-plantar erythrodysesthesia, and vomiting.—Hina Porcelli
Source: US Food and Drug Administration. FDA approves ripretinib for advanced gastrointestinal stromal tumor. May 15, 2020. www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-ripretinib-advanced-gastrointestinal-stromal-tumor. Accessed May 15, 2020.
