Merck, a leading science and technology company, announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s new drug application (NDA) for pimicotinib as a systemic treatment for patients with tenosynovial giant cell tumor (TGCT). The application is based on the primary results and longer-term follow-up of the global Phase 3 MANEUVER study, which demonstrated deep and durable tumor responses and meaningful improvements in clinical outcomes 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. “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.”
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The application is based on primary and longer-term results from the global Phase 3 MANEUVER study. In this trial, once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate (ORR) assessed by blinded independent review committee (BIRC) by RECIST v1.1 compared with placebo at week 25. The study also demonstrated statistically significant and clinically meaningful improvements in all secondary endpoints related to key patient-reported outcomes in TGCT, including improvements in active range of motion and physical function and reductions in stiffness and pain. These findings were presented at the 2025 ASCO Annual Meeting. Longer-term results with median follow-up of 14.3 months presented at ESMO Congress 2025 showed ORR continued to increase over time among patients treated with pimicotinib from the beginning of the study.
TGCT is a rare, locally aggressive tumor occurring in or around the joint leading to progressive swelling, stiffness and reduced mobility of the affected joint, significantly impacting daily activities and quality of life in what is typically an otherwise healthy population. If left untreated or in recurrent cases, TGCT may result in irreversible damage to the bone, joint and surrounding tissues. A significant need remains for highly effective and well-tolerated treatments beyond surgery that can not only shrink tumors but also alleviate pain and restore function.
In December 2025, pimicotinib was approved by the China National Medical Products Administration (NMPA) for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause functional limitation or relatively severe morbidity. Additional applications are under review by regulatory bodies in other markets.
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Source – businesswire
