Ono Pharmaceutical has filed for approval of ripretinib in Japan for advanced gastrointestinal stromal tumor based on the Phase 3 INVICTUS study, where the therapy significantly improved progression-free survival compared with placebo.
Written By: Pharmacally Medical News Desk
Ono Pharmaceutical has submitted an application in Japan for the manufacturing and marketing approval of ripretinib (DCC-2618), an oral tyrosine kinase inhibitor developed by Deciphera Pharmaceuticals, for the treatment of gastrointestinal stromal tumor (GIST) that has progressed following prior cancer chemotherapy.
The regulatory submission is supported by results from the global Phase 3 INVICTUS study (NCT03353753), which evaluated ripretinib in patients with advanced GIST who had previously received treatment with three or more kinase inhibitors, including imatinib. The randomized, double-blind, placebo-controlled study enrolled 129 patients across multiple international centers and compared once-daily ripretinib (150 mg) with placebo.
In the trial, ripretinib demonstrated a significant improvement in the primary endpoint of progression-free survival (PFS). Patients treated with ripretinib achieved a median PFS of 6.3 months, compared with 1.0 month for those receiving placebo, corresponding to a hazard ratio of 0.15 (p<0.0001) based on independent central radiologic review.
Tatsuya Okamoto, Corporate Officer and Executive Director of Clinical Development at Ono Pharmaceutical, said the submission represents an important step toward expanding treatment options for patients with GIST in Japan, a rare tumor worldwide. He noted that ripretinib has already been approved in more than 40 countries and regions, including the United States and Europe.
Ryota Udagawa, President and CEO of Deciphera Pharmaceuticals, added that the filing brings the therapy closer to patients in Japan with advanced GIST, highlighting the need to address delayed access to innovative medicines.
Ripretinib is designed to broadly inhibit KIT and PDGFRα mutations that drive the development of GIST. The drug targets multiple primary and secondary KIT mutations across exons 9, 11, 13, 14, 17, and 18, as well as PDGFRα mutations including exon 18 D842V, which are known to contribute to disease progression.
In Japan, ripretinib recently received orphan drug designation from the Ministry of Health, Labour and Welfare on March 19, 2026, for the treatment of GIST that has progressed following chemotherapy. The therapy has also been recognized as a drug addressing high unmet medical need by Japan’s Evaluation Committee on Unapproved or Off-label Drugs with High Medical Needs.
GIST is a rare mesenchymal tumor arising in the gastrointestinal tract, with an incidence of roughly 1–2 cases per 100,000 people annually. In Japan, approximately 1,492 new cases are diagnosed each year. Current approved treatments for unresectable or metastatic GIST include imatinib, sunitinib, regorafenib, and pimitespib as sequential lines of therapy.
References
Ono Pharma Submits an Application for Approval of Ripretinib (DCC-2618) in Patients with Advanced Gastrointestinal Stromal Tumor in Japan, 26 March 2026, Ono Pharma Submits an Application for Approval of Ripretinib (DCC-2618) in Patients with Advanced Gastrointestinal Stromal Tumor in Japan | ONO CORPORATE
Phase 3 Study of DCC-2618 vs Placebo in Advanced GIST Patients Who Have Been Treated with Prior Anticancer Therapies (INVICTUS), ClinicalTrials.gov ID NCT03353753, https://clinicaltrials.gov/study/NCT03353753