European Commission Approves Vimseltinib for Treatment of Tenosynovial Giant Cell Tumor

Deciphera's vimseltinib has received approval from the European Commission for the treatment of symptomatic tenosynovial giant cell tumor (TGCT) in adults in the European Union, providing a new therapeutic option for patients with limited surgical options. This approval is based on the compelling efficacy and safety results from the pivotal Phase 3 MOTION study and the Phase 1/2 study of vimseltinib. The treatment is now the first and only therapy with marketing authorization for TGCT in the European Union.

Key Takeaways:

• The European Commission has approved vimseltinib for the treatment of symptomatic tenosynovial giant cell tumor (TGCT) in adults in the European Union, providing a new therapeutic option for patients with limited surgical options.
• Vimseltinib is the first and only therapy with marketing authorization for TGCT in the European Union.
• The approval is based on the compelling efficacy and safety results from the pivotal Phase 3 MOTION study and the Phase 1/2 study of vimseltinib.
• The MOTION study demonstrated statistically significant and clinically meaningful improvements in active range of motion, patient-reported physical functioning, and patient-reported pain in patients with TGCT treated with vimseltinib compared to placebo.
• The safety profile of vimseltinib is manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial.
• Deciphera will leverage its global commercial infrastructure to bring vimseltinib to patients in the European Union.
• The approval follows the European Commission's recognition of the need for a new therapeutic option for TGCT patients, who often experience significant disability and pain.
• Jean-Yves Blay, M.D., Ph.D., highlighted the welcome news for the TGCT community, as vimseltinib is now the first approved therapy for TGCT in Europe.

Statistics:

• 23% (n=19/83) of 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 months.
• The Phase 3 MOTION study demonstrated statistically significant and clinically meaningful improvements in 6 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 pain.
• The safety profile of vimseltinib was manageable and consistent with results previously disclosed in the Phase 1/2 clinical trial.

Sources:

• Ono Pharmaceutical Co., Ltd. [1]
• Gelderblom H, Bhadri V, Stacchiotti S, et al. Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024;403(10445):2709-2719. doi:10.1016/S0140-6736(24)00885-7
• Stacchiotti S, DÈ­rr HR, Schaefer IM, et al. Best clinical management of tenosynovial giant cell tumour (TGCT): A consensus paper from the community of experts. Cancer Treat Rev. 2023;112:102491. doi:10.1016/j.ctrv.2022.102491