Ono Pharma Files Application in Japan for Ripretinib (DCC-2618) to Treat Advanced Gastrointestinal Stromal Tumors
Ono Pharmaceutical, headquartered in Osaka, Japan, under the leadership of President Toichi Takino, has announced the submission of a regulatory application in Japan for the manufacturing and marketing approval of ripretinib (DCC-2618). This investigational therapy, developed by Deciphera Pharmaceuticals, is intended for the treatment of patients with gastrointestinal stromal tumor (GIST) that has progressed after prior cancer chemotherapy. The submission marks an important step toward making this advanced treatment available to Japanese patients living with this rare and challenging form of cancer.
The approval request is based on data from the global Phase 3 INVICTUS study, which evaluated the efficacy and safety of ripretinib in adults with advanced GIST who had previously received treatment with at least three kinase inhibitors, including imatinib, the standard first-line therapy. In the study, ripretinib demonstrated a significant improvement in progression-free survival (PFS) compared to placebo, establishing its potential as a meaningful treatment option for patients whose tumors have become resistant to multiple prior therapies.
“GIST is classified as a rare disease worldwide, and ripretinib, developed by Deciphera, has already received approval in more than 40 countries and regions, including the United States and Europe. Our application in Japan represents an important step forward for patients who have exhausted prior therapies,” said Tatsuya Okamoto, Corporate Officer and Executive Director of Clinical Development at Ono. “We remain committed to the development and provision of innovative therapies that address unmet medical needs and contribute to society’s expectations for advanced healthcare solutions.”
Gastrointestinal stromal tumors, while rare, present unique treatment challenges. Patients with advanced GIST often face limited options after failing standard-of-care therapies, and delays in accessing novel treatments can exacerbate anxiety and compromise outcomes. Ripretinib’s global clinical development program has shown that it can provide clinically meaningful benefits, including extended disease control for patients with few remaining options.
“The submission of ripretinib in Japan brings us closer to providing patients with advanced GIST a potential new treatment avenue,” said Ryota Udagawa, President and CEO of Deciphera. “Access to innovative medicines remains a critical issue in Japan, especially for rare cancers where therapeutic options are limited. By offering new treatment possibilities, we hope to address some of the delays and gaps currently faced by patients and healthcare providers.”
Ripretinib’s mechanism of action targets multiple kinase mutations commonly associated with resistance in GIST, including those that emerge after treatment with earlier-generation inhibitors. This broad-spectrum inhibition is designed to control tumor progression more effectively and prolong the time patients can remain on therapy without disease worsening. By offering an additional line of defense in the treatment sequence, ripretinib has the potential to improve both patient outcomes and quality of life.
Both Ono and Deciphera emphasized their shared commitment to ensuring timely patient access. The companies are actively preparing for regulatory review and working to facilitate rapid availability should approval be granted. Their joint efforts underscore the importance of collaboration between local and global biopharmaceutical organizations in accelerating access to innovative therapies for rare diseases.
“In rare tumors such as GIST, patients and caregivers often face significant uncertainty due to the scarcity of information and limited therapeutic choices. Our goal is to reduce this uncertainty by providing access to ripretinib as efficiently as possible,” Udagawa added. “Together with our partner companies, we are dedicated to delivering this treatment to Japanese patients with advanced GIST in a timely manner.”
The submission in Japan follows the broader global adoption of ripretinib, which has been approved in multiple regions based on its demonstrated efficacy and safety profile. As a next-generation tyrosine kinase inhibitor, it has been recognized for its ability to manage advanced GIST with complex resistance patterns, providing hope for patients who previously had few remaining options.
Ono Pharmaceutical continues to focus on innovative oncology therapies and the expansion of its portfolio of rare disease treatments. By collaborating with Deciphera and leveraging global clinical expertise, Ono aims to address significant unmet needs in Japan’s oncology landscape while advancing the availability of life-changing therapies to patients with limited treatment alternatives.
Looking ahead, the companies will engage with Japan’s regulatory authorities to ensure a thorough review process while maintaining patient-centric priorities. Should the application be approved, ripretinib will become a critical addition to the treatment armamentarium for GIST in Japan, complementing existing therapies and providing a new option for patients in advanced stages of the disease.
In summary, the submission of ripretinib by Ono Pharmaceutical represents a pivotal milestone for Japanese patients with advanced GIST. Supported by robust Phase 3 clinical data from the INVICTUS study and global regulatory experience, this effort reflects a commitment to accelerating access to innovative therapies, improving patient outcomes, and addressing the challenges associated with rare cancers in Japan. Both Ono and Deciphera remain focused on ensuring that patients in need can benefit from ripretinib as promptly as possible.
About the INVICTUS Study
The INVICTUS Phase 3 clinical study is a randomized, double-blind, placebo-controlled, international, multicenter study conducted in 12 overseas countries including US and EU to evaluate the safety, tolerability, and efficacy of ripretinib compared to placebo in 129 patients with advanced GIST who have received prior treatment with 3 or more kinase inhibitors, including imatinib. Patients were randomized 2:1 to either 150 mg of ripretinib or placebo once daily.
In the INVICTUS study, ripretinib significantly prolonged the primary endpoint of PFS determined by independent central radiologic review (a median PFS of 6.3 months in ripretinib arm compared to 1.0 month in the placebo arm, HR of 0.15, p<0.0001). 1)
About GIST
GIST is a mesenchymal tumor that arises from the muscular layer of the gastrointestinal tract and forms a mass that pushes up the mucosa from below. GIST is a rare disease, with an incidence of approximately 1 to 2 cases per 100,000 population per year. In Japan, the number of patients diagnosed with GIST between 2016 and 2018 was 4,475 over three years (approximately 1,492 cases per year; crude incidence rate: 1.18 cases per 100,000 population). 2) It is known that mutations in KIT (KIT proto-oncogene receptor tyrosine kinase) and PDGFRα (platelet-derived growth factor receptor α) are involved in the development of the majority of patients with GIST.
For unresectable, metastatic, or recurrent GIST, four agents—imatinib, sunitinib, regorafenib, and pimitespib—are currently approved in Japan. According to the Japanese clinical practice guidelines for GIST, these agents are recommended as first-line, second-line, third-line, and fourth-line treatments, respectively. 3)
About ripretinib
Ripretinib is an orally administered, tyrosine kinase inhibitor developed by Deciphera. Ripretinib inhibits primary and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18, which are involved in GIST. In addition, ripretinib inhibits Pharma primary PDGFRα mutations in exons 12, 14, and 18, including the exon 18 D842V mutation, which is present in a subset of GIST.
The INVICTUS study has demonstrated the significant efficacy of ripretinib in patients with advanced GIST who have received prior treatment with 3 or more kinase inhibitors, including imatinib. Ripretinib was launched in the U.S. in 2020 under the brand name QINLOCK®. As of March 2026, it is approved for the treatment of advanced GIST in more than 40 countries or regions, including the U.S. and European countries.
In Japan, ripretinib was designated as an orphan drug by the Ministry of Health, Labour and Welfare on March 19, 2026, for the treatment of GIST that have progressed following cancer chemotherapy. In addition, ripretinib was determined as a drug with high medical needs at the “67th Evaluation Committee on Unapproved or Off-label Drugs with High Medical Needs” held by Ministry of Health, Labour and Welfare, aiming to eliminate drug losses in Japan.
About Ono Pharmaceutical Co., Ltd.
Ono Pharmaceutical Co., Ltd. delivers innovative therapies for patients worldwide. Upholding its philosophy of “Dedicated to the Fight against Disease and Pain,” Ono targets areas with unmet medical needs including oncology, immunology & inflammation, and neurology, and fosters partnerships with academic and biotech organizations to accelerate Pharma drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company’s website at https://www.ono-pharma.com/en.
About Deciphera Pharmaceuticals Inc.
Deciphera, a member of Ono Pharmaceutical Co., Ltd., is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines and providing hope to people living with cancer, neurologic and autoimmune disease. Deciphera is leveraging its proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines.
In addition to advancing multiple product candidates from Deciphera’s platform in clinical studies, QINLOCK® (ripretinib) is Deciphera’s switch-control kinase inhibitor approved in many countries including the European Union and the United States for the treatment of adult patients with advanced GIST who have received prior treatment with 3 or more kinase inhibitors, including imatinib.
ROMVIMZA® is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity, and in the Pharma European Union for adult patients with TGCT associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability.
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