On September 17, 2025 Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono"), reported that the European Commission (EC) has approved ROMVIMZA (vimseltinib) in the European Union (EU) for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability (Press release, Deciphera Pharmaceuticals, SEP 17, 2025, View Source [SID1234656040]).
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"The European Commission’s approval of vimseltinib for TGCT is a significant milestone for Deciphera, ONO, and TGCT patients across the European Union who are in need of a non-invasive treatment option. We are excited to leverage our global commercial infrastructure to bring vimseltinib to these patients," said Ryota Udagawa, President and Chief Executive Officer of Deciphera. "We look forward to working with health authorities to ensure all eligible patients who can benefit from vimseltinib have access as quickly as possible."
"This is welcome news for the TGCT community as vimseltinib is now the first approved therapy for TGCT in Europe," said Jean-Yves Blay, M.D., Ph.D., Leon Berard Center. "TGCT can significantly impact the daily lives of patients by causing pain, stiffness and mobility limitations. Vimseltinib is a differentiated treatment that has demonstrated the ability to address these unmet patient needs while remaining well-tolerated."
The EC approval is supported by compelling efficacy and safety results from the pivotal Phase 3 MOTION study of vimseltinib in patients with TGCT not amenable to surgery with no prior anti-CSF1/CSF1R therapy (prior therapy with imatinib or nilotinib allowed), compared to placebo, as well as the Phase 1/2 study of vimseltinib1. The primary endpoint was supported by statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm compared to the placebo arm at week 251. The secondary endpoint was supported by statistically significant and clinically meaningful improvements versus placebo in all six key secondary endpoints assessed at Week 25 including objective response rate (ORR) by tumor volume score (TVS), active range of motion (ROM), physical function, stiffness, quality of life, and pain1. In a descriptive analysis at Week 97, 23% (n=19/83) of the patients randomized to receive vimseltinib had best overall response of complete response (CR) according to RECIST v1.1, as assessed by blind independent radiological review (IRR), with a median time to CR of 11.5 months1. The safety profile of vimseltinib is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial1. For a full list of side effects and information on dosage and administration and other precautions, please refer to the Summary of Product Characteristics for further information.
About Tenosynovial Giant Cell Tumor (TGCT)
TGCT is caused by a translocation in colony-stimulating factor 1 (CSF1) gene resulting in overexpression of CSF1 and recruitment of colony-stimulating factor 1 receptor (CSF1R)-positive inflammatory cells into the lesion.2 TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS). TGCT is a rare, locally aggressive neoplasm that can grow and cause damage to surrounding tissues and structures inducing pain, swelling, and limitation of movement of the joint. Surgery is the main treatment option; however, these tumors tend to recur, particularly in diffuse-type TGCT. If untreated or if the tumor continually recurs, damage and degeneration may occur in the affected joint and surrounding tissues, which may cause significant disability. For a subset of patients, surgical resection will potentially cause worsening functional limitation or severe morbidity, systemic treatment options are limited and a new therapeutic option for TGCT is needed.