European Commission Green-Lights Vimseltinib for Symptomatic, Unresectable TGCT

The European Commission (EC) has approved vimseltinib (Romvimza) for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) associated with clinically relevant physical function deterioration and for whom surgical options have been exhausted or would induce unacceptable morbidity or disability.1

The approval was supported by data from the phase 3 MOTION trial (NCT05059262) and a phase 1/2 trial (NCT03069469). Of note, the MOTION study evaluated the efficacy and safety of vimseltinib for the treatment of patients with TGCT not amenable to surgery who had not previously received anti-CSF1/CSF1R therapy. However, patients on the study were permitted to have prior treatment of imatinib (Gleevec) or nilotinib (Tasigna). Moreover, the phase 1/2 study evaluated the safety, efficacy, and pharmacokinetics of vimseltinib for the treatment of patients with malignant solid tumors and TGCT.2

“The EC’s approval of vimseltinib for TGCT is a significant milestone for Deciphera, ONO [Pharmaceutical], and [patients with] TGCT 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,” Ryota Udagawa, president and CEO of Deciphera, stated in a news release.1 “We look forward to working with health authorities to ensure all eligible patients who can benefit from vimseltinib have access as quickly as possible.”

What Were the Efficacy and Safety Data From the MOTION Study?

At week 97 in a descriptive analysis, 23% of patients (n = 19 of 83) who were randomly assigned to receive vimseltinib achieved the best overall response of complete response (CR) per RECIST 1.1 criteria by blind independent radiological review, with a median time to CR of 11.5 months.

Furthermore, the safety profile of vimseltinib was manageable and consistent with previous data produced during the phase 1/2 study.

What Was the MOTION Study Design?

Patients enrolled in the phase 3 study were at least 18 years of age with tumor biopsy–confirmed, unresectable TGCT that was symptomatic, as defined as at least moderate pain or moderate stiffness.3 Additionally, patients needed to receive a stable analgesic regimen for at least 2 weeks before receiving the first dose of the study treatment. Common inclusion criteria also included measurable disease per RECIST 1.1 criteria with at least 1 lesion of at least 2 cm, as well as adequate organ and bone marrow function.

The multicenter study consisted of 2 parts: patients in part 1 are randomly assigned to receive either vimseltinib or a matching placebo for 24 weeks; patients in part 1 had the option to be treated with vimseltinib for part 2, which is a long-term treatment phase. Specifically, patients in parts 1 and 2 receiving vimseltinib were treated with blinded treatment of 30 mg twice a week for 24 weeks in part 1 and open-label vimseltinib at 30 mg twice a week in part 2. Those receiving placebo received blinded treatment twice a week for 24 weeks in part 1 and open-label vimseltinib at 30 mg twice a week in part 2.

The primary end point of the study was objective response rate (ORR) per RECIST 1.1 criteria. Secondary end points include ORR at week 25 per tumor volume score, active range of motion at week 25, physical function, changes in stiffness, pain, and quality of life. Notably, the primary end point was supported by improvements that were statistically significant and clinically meaningful regarding range of motion, patient-reported physical functioning, and pain in the vimseltinib vs placebo arms at week 251.1 The 6 respective secondary end points were also supported by statistically significant and clinically meaningful improvements.

“This [approval of vimseltinib] is welcome news for the TGCT community as vimseltinib is now the first approved therapy for TGCT in Europe,” Jean-Yves Blay, MD, PhD, of the Léon Bérard Center in Lyon, France, added in the news release. “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.”

References

1. Deciphera receives European Commission Approval of Romvimza (vimseltinib) for the treatment of tenosynovial giant cell tumor (TGCT). News release. Deciphera. September 17, 2025. Accessed September 18, 2025. https://www.deciphera.com/news/deciphera-receives-european-commission-approval-for-romvimza
2. Study of vimseltinib (DCC-3014) in patients with advanced tumors and tenosynovial giant cell tumor. ClinicalTrials.gov. Updated November 20, 2024. Accessed September 18, 2025. https://www.clinicaltrials.gov/study/NCT03069469
3. Study of vimseltinib for tenosynovial giant cell tumor (MOTION). ClinicalTrials.gov. Updated February 24, 2025. Accessed September 18, 2025. https://clinicaltrials.gov/study/NCT05059262