FDA Accepts New Drug Application for Pimicotinib in Tenosynovial Giant Cell Tumor
Key Clinical Takeaways:
- The FDA has accepted the New Drug Application for pimicotinib based on results from the global, randomized, double-blind, placebo-controlled phase 3 MANEUVER trial evaluating once-daily oral pimicotinib in patients with tenosynovial giant cell tumor.
- Pimicotinib significantly improved objective response rate at week 25 compared with placebo, with responses assessed by blinded independent review using RECIST v1.1. Improvements were also observed across secondary patient-reported outcomes, including range of motion, physical function, stiffness, and pain.
- If approved, pimicotinib would represent an oral systemic treatment option for patients with symptomatic tenosynovial giant cell tumor, a locally aggressive disease associated with substantial morbidity and limited nonsurgical therapies.
Based on efficacy and safety findings from the phase 3 MANEUVER trial, the US Food and Drug Administration (FDA) has formally accepted the New Drug Application for pimicotinib, an oral colony-stimulating factor 1 receptor (CSF-1R) inhibitor, for the treatment of patients with tenosynovial giant cell tumor (TGCT).
In this multicenter, double-blind, placebo-controlled study, 90 patients were randomized 2:1 to receive 50 mg of pimicotinib or placebo once daily. Following 24 weeks of treatment, patients randomized into the placebo arm were permitted to either continue placebo or switch into the pimicotinib for an additional 24 weeks. The primary end point was objective response rate (ORR), as assessed via blinded independent review. Key secondary end points included ORR by tumor volume score, change in clinical outcome assessments, and safety.
At initial analysis, ORR was 54% in the pimicotinib arm and 3.2% in the placebo arm (P <.001), and ORR by tumor volume score was 61.9% and 3.2%, respectively (P <.0001). At longer-term follow-up (62 months), ORR increased to 76.2% and ORR by tumor volume score increased to 74.6% in patients initially randomized into the pimicotinib arm. Pimicotinib significantly improved outcomes across all clinical outcome assessments including active range of movement, physical function, and reductions in stiffness and pain.
Safety was consistent with that of CSF-1R inhibitors, and most treatment-emergent adverse events were low grade. Dose reductions occurred in 7.9% of patients, and treatment discontinuations were reported in 1.6%. No evidence of cholestatic hepatotoxicity or drug-induced liver injury was observed.
“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.
