Vimseltinib Earns Positive CHMP Recommendation for TGCT

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending the approval of vimseltinib (Romvimza) 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.1

The positive opinion was supported by data from the phase 3 MOTION trial (NCT05059262), along with findings from a phase 1/2 study (NCT03069469). In MOTION, findings showed that patients treated with vimseltinib experienced a statistically significant and clinically meaningful improvement in objective response rate (ORR) at week 25 per blinded independent radiologic review (BIRR) according to RECIST 1.1 criteria. The 25-week ORRs were 40% for vimseltinib vs 0% for placebo (P < .0001).

Additionally, statistically significant and clinically meaningful improvements were also observed in the vimseltinib arm regarding active range of motion, patient-reported physical functioning, and patient-reported pain observed at week 25.

The safety profile of vimseltinib is manageable and consistent with previously reported data.

A final decision on approval from the European Commission is expected by the end of September 2025.

“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,” Ryota Udagawa, president and chief executive officer of Deciphera Pharmaceuticals, stated in a news release. “We look forward to building upon vimseltinib’s positive CHMP opinion as we work to bring this medicine to [patients with] TGCT around the world in need of new treatment options.”

In February 2025, the FDA approved vimseltinib for the treatment of patients with symptomatic TGCT for whom surgical resection will potentially cause worsening functional limitation or severe morbidity.2 This regulatory decision was also supported by findings from MOTION.

MOTION Overview

The randomized, placebo-controlled, double-blind trial enrolled patients at least 18 years of age with TGCT who were ineligible for surgical resection.3 Patients were required to have symptomatic disease, defined as at least moderate pain or at least moderate stiffness. Patients needed to complete 14 consecutive days of questionnaires during the screening period and were required to meet minimum thresholds within the study protocol. Other key inclusion criteria comprised a stable analgesic regimen for at least 2 weeks prior to first dose of study drug, per physician discretion; measurable disease with at least 1 lesion with a minimum size of 2 cm per RECIST 1.1 criteria; and adequate organ and bone marrow function.

Patients who previously received systemic CSF1- or CSF1R-targeted therapy were excluded; however, prior treatment with imatinib (Gleevec) and nilotinib (Tasigna) was allowed. Other exclusion criteria included therapy for TGCT, including investigational therapy, during the screening period; participation in a non-TGCT investigational drug study within 30 days of screening; and known metastatic TGCT or other active cancer requiring concurrent treatment.

In part 1 of the study, patients were randomly assigned to receive blinded treatment with 30 mg of vimseltinib twice per week or matching placebo for 24 weeks; in part 2, all patients were administered open-label vimseltinib at 30 mg twice per week.

ORR at week 25 served as the trial’s primary end point. Secondary end points included ORR at week 25 per tumor volume score, change in active range of motion, change in physical function, change in stiffness rating, and change in pain among responders.

References

1. Deciphera announces positive CHMP opinion for Romvimza (vimseltinib) for the treatment of tenosynovial giant cell tumor (TGCT). News release. Deciphea Pharmaceuticals. July 28, 2025. Accessed July 28, 2025. https://www.deciphera.com/news/deciphera-announces-positive-chmp-opinion-romvimzatm-vimseltinib-treatment-tenosynovial-giant
2. FDA approves vimseltinib for symptomatic tenosynovial giant cell tumor. FDA. February 14, 2025. Accessed July 28, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-vimseltinib-symptomatic-tenosynovial-giant-cell-tumor
3. Study of vimseltinib for tenosynovial giant cell tumor (MOTION). ClinicalTrials.gov. Updated February 24, 2025. Accessed July 28, 2025. https://clinicaltrials.gov/study/NCT05059262