European Commission approves vimseltinib for treatment of TGCT

September 2025 – The European Commission approved vimseltinib for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT) not qualifying for surgery and with clinically relevant deterioration of physical function. This is the 1st drug in Europe approved for treatment of TGCT, an important milestone for European patients.

Based on MOTION Phase 3 Study

The application is supported by results from the Phase 3 MOTION study, where vimseltinib showed significant improvements compared to placebo:

• Objective Response Rate (ORR) at Week 25: 40% in the vimseltinib group vs. 0% in placebo (p <0.0001).
• Clinically meaningful improvements were also seen in secondary endpoints, including tumor volume, range of motion, physical function, stiffness, quality of life, and pain.

The safety profile of vimseltinib was considered manageable and consistent with earlier trials. Results were presented at the 2024 ASCO Annual Meeting.

Addressing an Unmet Need

TGCT is a rare, non-malignant tumor that develops in or near joints, caused by overproduction of CSF1. While not cancerous, TGCT can be aggressive, leading to joint damage, pain, and significant disability. Surgery is the main treatment, but tumors often recur, particularly in diffuse-type TGCT. For patients not eligible for surgery, there are currently very few systemic treatment options.

Orphan Drug Designation

Vimseltinib was granted Orphan Drug Designation by the EMA in December 2019, recognizing the need for innovative treatments for rare diseases like TGCT.

This regulatory milestone brings hope that vimseltinib could soon offer a much-needed treatment option for TGCT patients in Europe who currently face limited alternatives.