BOSTON, Mass. — The U.S. Food and Drug Administration has accepted a new drug application from EMD Serono for pimicotinib as a systemic treatment for patients with tenosynovial giant cell tumor, a rare and locally aggressive disease that affects joints and surrounding tissue.
EMD Serono, the healthcare business of Merck KGaA, Darmstadt, Germany, said the application is supported by primary and longer-term follow-up data from the global Phase 3 MANEUVER clinical trial, which demonstrated deep and durable tumor responses as well as meaningful improvements in clinical outcomes among patients treated with pimicotinib.
“With pimicotinib, we have an opportunity to significantly advance care for people living with TGCT, a painful and debilitating disease that has few effective and well-tolerated treatment options beyond surgery,” said David Weinreich, Global Head of R&D and Chief Medical Officer for the Healthcare business of Merck KGaA, Darmstadt, Germany. “Based on clinical trial results showing not only a reduction in tumor burden, but also the ability to help alleviate symptoms like pain and stiffness in the global Phase 3 MANEUVER study, we are confident in the important role pimicotinib can play for TGCT patients in the U.S. and worldwide.”
In the MANEUVER study, once-daily pimicotinib met the primary endpoint by demonstrating a statistically significant improvement in objective response rate, as assessed by a blinded independent review committee using RECIST v1.1 criteria, compared with placebo at week 25. The trial also showed statistically significant and clinically meaningful improvements across all key secondary endpoints, including patient-reported measures of active range of motion, physical function, stiffness, and pain.
The results were presented at the 2025 ASCO Annual Meeting. Longer-term findings with a median follow-up of 14.3 months, presented at the 2025 ESMO Congress, showed that objective response rates continued to increase over time among patients who received pimicotinib from the start of the study.
Tenosynovial giant cell tumor is a rare condition that develops in or around joints and can cause progressive swelling, stiffness, and reduced mobility, significantly affecting daily activities and quality of life. In untreated or recurrent cases, the disease can lead to irreversible damage to bone, joints, and surrounding tissue. While surgery remains a primary treatment option, there is an ongoing need for effective and well-tolerated systemic therapies that can reduce tumor burden while improving symptoms and function.
In December 2025, pimicotinib was approved by China’s National Medical Products Administration for the treatment of adult patients with symptomatic TGCT for whom surgical resection may result in functional limitations or significant morbidity. Regulatory reviews are ongoing in additional markets.
