FDA Accepts New Drug Application for Pimicotinib in Symptomatic Tenosynovial Giant Cell Tumor
Clinical Summary:
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The FDA has accepted the New Drug Application for pimicotinib based on findings from the phase 3 MANEUVER trial evaluating the oral colony-stimulating factor 1 receptor (CSF-1R) inhibitor in patients with symptomatic tenosynovial giant cell tumor (TGCT).
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In this double-blind, placebo-controlled trial, pimicotinib significantly improved objective response rate compared with placebo and demonstrated clinically meaningful improvements in range of motion, physical function, stiffness, and pain. The safety profile was consistent with previous studies, with most treatment-emergent adverse events being low grade.
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If approved, pimicotinib could provide a once-daily oral treatment option for patients with symptomatic TGCT, expanding nonsurgical treatment options for this locally aggressive disease.
The US Food and Drug Administration (FDA) has accepted the New Drug Application for pimicotinib, an investigational oral colony-stimulating factor 1 receptor (CSF-1R) inhibitor, for the treatment of patients with symptomatic tenosynovial giant cell tumor (TGCT). The application is supported by results from the phase 3 MANEUVER trial.
In this global, randomized, double-blind, placebo-controlled trial, 90 patients were randomized 2:1 to receive 50 mg of once daily pimicotinib or placebo. After 24 weeks, patients assigned to placebo could either continue placebo or cross over to pimicotinib for an additional 24 weeks. The primary end point was objective response rate (ORR), as assessed by blinded independent review. Key secondary end points included tumor volume score response, patient-reported outcomes, and safety.
At the primary analysis, ORR was 54% with pimicotinib compared with 3.2% with placebo (P < .001). Objective response assessed by tumor volume score was 61.9% and 3.2%, respectively (P < .0001). With longer follow-up, ORR increased to 76.2% among patients initially assigned to pimicotinib, while tumor volume score response increased to 74.6%.
Pimicotinib also produced clinically meaningful improvements across multiple patient-reported outcomes, including active range of motion, physical function, stiffness, and pain.
The safety profile was consistent with that of other CSF-1R inhibitors. Most treatment-emergent adverse events were grade 1/2. Dose reductions occurred in 7.9% of patients, treatment discontinuation occurred in 1.6%, and no cases of cholestatic hepatotoxicity or drug-induced liver injury were reported.
“With regulatory submissions progressing across major markets worldwide, pimicotinib is expected to provide TGCT patients with a once-daily, oral, effective and well-tolerated therapeutic option, helping address unmet clinical needs in the management of TGCT.”
Sources:
PR Newswire. Abbisko therapeutics announces FDA acceptance of the NDA for pimicotinib for the treatment of tenosynovial giant cell tumor. Accessed February 5, 2026. https://www.prnewswire.com/news-releases/abbisko-therapeutics-announces-fda-acceptance-of-the-nda-for-pimicotinib-for-the-treatment-of-tenosynovial-giant-cell-tumor-302659104.html
Niu X, Ravi V, Broto JM, et al. Pmicotinib in tenosynovial giant cell tumor (TGCT): Efficacy, safety and patient-reported outcomes of phase 3 MANEUVER study. Presented at the 2025 ASCO Annual Meeting. May 30 - June 5, 2025; Chicago, Illinois. doi:10.1200/JCO.2025.43.16_suppl.11500
Niu X, Gelderblom H, Ravi V, et al. Extended efficacy and safety from the phase III MANEUVER trial of pimicotinib in patients with tenosynovial giant cell tumor (TGCT). Presented at the 2025 ESMO Congress. October 17-21, 2025; Berlin, Germany. Abstract 2690MO.
