On July 28, 2025 Deciphera Pharmaceuticals, LLC, a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines, and Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono"), reported that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the approval of vimseltinib 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, JUL 28, 2025, View Source [SID1234654587]).
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The positive CHMP opinion is a scientific recommendation for marketing authorization and one of the final steps before the European Commission (EC), which has the authority to approve medicines in the European Union (EU), issues a decision on Deciphera’s marketing authorization application (MAA) for vimseltinib. This decision is expected in the second quarter of the fiscal year ending March 31, 2026.
"CHMP’s positive opinion is an important milestone for the TGCT community in the European Union (EU), where there are currently no approved treatments for TGCT, and for vimseltinib, which is now one step closer to potential EU regulatory approval," said Ryota Udagawa, President and Chief Executive Officer of Deciphera. "We look forward to building upon vimseltinib’s positive CHMP opinion as we work to bring this medicine to TGCT patients around the world in need of new treatment options."
The positive CHMP opinion 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 vimseltinib. In MOTION, vimseltinib demonstrated a statistically significant and clinically meaningful objective response rate (ORR) at Week 25 in the intent-to-treat (ITT) population, as assessed by blinded independent radiologic review (BIRR) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1), versus placebo (40% in vimseltinib arm vs 0% in placebo arm, p <0.0001). 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 25. The safety profile of vimseltinib is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial.
About vimseltinib
Vimseltinib is an oral switch-control tyrosine kinase inhibitor specifically designed to selectively and potently inhibit CSF1R. Vimseltinib has been developed using Deciphera’s proprietary switch-control kinase inhibitor platform. ROMVIMZA (vimseltinib) 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.
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. TGCT is a rare, non-malignant tumor that develops inside or near joints. TGCT is caused by dysregulation of the CSF1 gene leading to overproduction of CSF1. TGCT is also known as giant cell tumor of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS), a diffuse-type of TGCT. TGCT is a 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.