Ono Seeks Approval for Ripretinib, Offering New Hope for GIST Patients in Japan

OSAKA, Japan – March 26, 2026 – Ono Pharmaceutical Co., Ltd. has submitted an application for the approval of ripretinib in Japan, a move that could provide a critical new treatment option for patients with an advanced and rare form of cancer. The application, announced today, seeks manufacturing and marketing approval for the drug to treat advanced gastrointestinal stromal tumor (GIST) that has progressed despite extensive prior therapies.

Developed by Deciphera Pharmaceuticals, now a subsidiary of Ono, ripretinib is a novel kinase inhibitor already approved in over 40 countries, including the United States and Europe, where it is marketed as QINLOCK®. The submission in Japan is a significant step toward addressing a major unmet medical need for a patient population with few remaining lifelines.

A Critical Lifeline for a Rare Cancer

Gastrointestinal stromal tumor, or GIST, is a rare sarcoma that arises in the digestive tract, affecting an estimated 1,500 new patients in Japan each year. While initial treatments can be effective, the disease presents a formidable challenge for patients with advanced or metastatic cases who develop resistance to standard therapies.

Currently, the treatment pathway in Japan for advanced GIST follows a well-defined sequence of kinase inhibitors: imatinib as a first-line therapy, followed by sunitinib and then regorafenib. For those who see their disease progress further, pimitespib was approved in 2022 as a fourth-line option. However, resistance is a persistent issue, driven by secondary mutations in the KIT and PDGFRα genes that fuel the cancer's growth. For patients who exhaust these options, the prognosis is often grim, leaving them and their families in a state of significant anxiety.

Ripretinib is designed specifically for this scenario. The Japanese application is supported by the pivotal Phase 3 INVICTUS study, a global, randomized, placebo-controlled trial. The results, published in The Lancet Oncology, were striking: patients treated with ripretinib had a median progression-free survival (PFS) of 6.3 months, compared to just 1.0 month for patients receiving a placebo. This represents an 85% reduction in the risk of disease progression or death, offering patients months of additional time before their cancer worsens.

“This application for approval of ripretinib represents a significant advancement for patients with GIST in Japan,” said Tatsuya Okamoto, Corporate Officer and Executive Director of Clinical Development at Ono, in a statement. He emphasized the company's commitment "to developing and providing innovative medicines to meet the treatment needs of patients."

Accelerating Access and Closing the 'Drug Lag'

The submission of ripretinib highlights a broader shift in Japan's pharmaceutical landscape. The country has historically contended with a "drug lag," where innovative medicines approved in the U.S. and Europe faced significant delays before becoming available to Japanese patients. In response, regulators have implemented several fast-track mechanisms to accelerate access, particularly for drugs addressing high unmet needs.

Ripretinib is a prime beneficiary of these initiatives. On March 19, 2026, Japan's Ministry of Health, Labour and Welfare (MHLW) granted it an orphan drug designation. This status is reserved for treatments for diseases affecting fewer than 50,000 people in Japan and offers incentives like priority review, R&D subsidies, and a potential 10-year market exclusivity period post-approval.

Furthermore, an MHLW committee identified ripretinib as a drug with high medical needs, specifically aiming to eliminate delays in its Japanese availability. These dual designations signal the drug's importance and suggest its review by the Pharmaceuticals and Medical Devices Agency (PMDA) could be expedited, potentially reducing the standard 12-month review period to around 9 months.

“The application submission brings us one step closer to providing patients in Japan with advanced GIST a potential new treatment option," stated Ryota Udagawa, President and Chief Executive Officer of Deciphera. "We anticipate that this submission may help address the issue of delayed access to new medicines, which remains a significant challenge for patients in Japan.”

A Strategic Cornerstone for Ono's Global Ambitions

For Ono Pharmaceutical, the ripretinib application is more than a regulatory milestone; it is a key validation of its recent corporate strategy. In June 2024, Ono completed a landmark $2.4 billion acquisition of Deciphera Pharmaceuticals, the U.S.-based developer of ripretinib. The move was a clear signal of Ono's intent to bolster its oncology portfolio and expand its global footprint.

The acquisition provided Ono with not only ripretinib, which generated $159.1 million in global sales in 2023, but also an established commercial infrastructure in the U.S. and Europe and a pipeline of other promising cancer therapies. Securing approval for ripretinib in its home market of Japan is a crucial step in realizing the full value of this major investment.

This strategic maneuver aligns with Ono's stated goal of becoming a "global specialty pharma" company by 2031, with oncology as a central pillar. By integrating Deciphera's kinase inhibitor expertise and commercial platforms, Ono is accelerating its transformation from a primarily Japan-focused company into a significant player on the world stage. The potential approval of ripretinib in Japan would not only provide a new revenue stream from a high-value market but also reinforce the company's reputation as a leader in delivering innovative cancer treatments.

As the PMDA begins its review, the hopes of many in the Japanese GIST community rest on a positive outcome. The potential arrival of ripretinib represents a convergence of scientific innovation, regulatory evolution, and strategic corporate vision, which could soon translate into more time and a better quality of life for patients battling this relentless disease.