FDA Accepts New Drug Application for Pimicotinib in Tenosynovial Giant Cell Tumour
Tuesday, January 13, 2026
Merck has announced that the US Food and Drug Administration (FDA) has accepted its new drug application for pimicotinib as a systemic treatment for patients with tenosynovial giant cell tumour (TGCT). The submission is supported by primary results and longer-term follow-up data from the global Phase 3 MANEUVER study.
The Phase 3 study showed that pimicotinib delivered deep and durable tumour responses, along with meaningful improvements in clinical outcomes. Patients treated with once-daily pimicotinib achieved a statistically significant improvement in the primary endpoint of objective response rate compared with placebo at week 25, as assessed by a blinded independent review committee using RECIST v1.1 criteria.
The study also met all key secondary endpoints, demonstrating clinically meaningful improvements in patient-reported outcomes. These included better active range of motion and physical function, as well as reductions in stiffness and pain. Initial results were presented at the 2025 ASCO Annual Meeting. Longer-term data, with a median follow-up of 14.3 months, were later presented at the 2025 ESMO Congress and showed that objective response rates continued to increase over time in patients who received pimicotinib from the start of the study.
TGCT is a rare and locally aggressive tumour that develops in or around joints. It can cause progressive swelling, stiffness and reduced joint movement, significantly affecting daily activities and quality of life. If not treated, or if it recurs, the disease can lead to permanent damage to joints, bones and surrounding tissues. There remains a clear need for effective and well-tolerated treatments beyond surgery that can reduce tumour size, relieve pain and restore function.
In December 2025, pimicotinib received approval from the China National Medical Products Administration for the treatment of adults with symptomatic TGCT where surgery may result in functional limitation or significant morbidity. Regulatory reviews are ongoing in other regions.
Source: businesswire.com
