According to ONCLive, ripretinib, designed to treat patients with advanced gastrointestinal stroll tumors (GIST), was given Priority Review status in China by the National Medical Products Administration. This status is designed to bring drugs with significant clinical value to the Chinese marketplace.
Gastrointestinal Stromal Tumors (GIST)
Gastrointestinal stromal tumors (GIST) generally result from spontaneous KIT or PDGFR gene mutations. These tumors develop in the digestive tract, usually in the stomach or small intestine. However, in rarer cases, GIST can form in the esophagus, rectum, and colon. As a soft-tissue sarcoma, GIST affects connective tissues around other bodily structures. When interstitial cells of Cajal (ICCs) experience DNA changes, a GIST appears. Generally, these tumors occur in patients between the ages of 40 and 70. Around 5,000 new cases are diagnosed in the U.S. each year, and up to 30,000 cases are diagnosed in China. Symptoms include:
- Bloody stool or bloody vomit
- Muscle weakness
- Unintended weight loss
- Appetite loss
- Nausea and vomiting
- Abdominal pain and swelling
Read more on GIST here.
According to Pharma’s Almanac, China’s priority review process seeks drugs with significant clinical value:
To qualify, novel drugs must meet at least one of several different criteria, which include never having been marketed in or outside China; using innovative technology or offering significant treatment advantages; having patent protection that will expire within 3 years for a clinical trial application or 1 year for a commercial application; [and] having the manufacturing transferred to China.
In this case, Priority Review status was granted to a new drug application for ripretinib, also known as Qinlock. The drug is designed for patients with advanced GIST who have previously been treated with at least 3 kinase inhibitors. Generally, kinase inhibitors block protein kinases from functioning. In this case, ripretinib inhibits KIT and PDGFRα–mutated kinases.
Related Data for Treating GIST
Prior to China’s Priority Review, ripretinib was approved by the FDA as a fourth-line treatment option for patients with advanced GIST. This approval was based on data from the Phase 3 INVICTUS trial, which compared the efficacy of ripretinib versus a placebo. Within this trial, ripretinib reduced disease progression and death by 85%. Further, the drug more than doubled the overall survival rate when compared to a placebo.
During the trial, 98.8% of patients taking ripretinib did experience adverse reactions. These included fatigue, nausea, alopecia, constipation, abdominal and muscle pain, anemia, and hypertension. As a result, some patients required lower doses.
Moving forward, an additional Phase 3 clinical trial (INTRIGUE) will explore the efficacy of ripretinib as compared to sunitinib. The trial, which hopes to finish in June 2021, will enroll 358 patients with advanced GIST.