Pimicotinib Earns Approval in China for Tenosynovial Giant Cell Tumors

Supporting data for the approval came from the phase 3 MANUEVER trial (NCT05804045), in which investigators assessed the safety and efficacy of pimicotinib among patients with TGCTs who received no prior anti–CSF-1 or anti–CSF-1R treatment.

China’s National Medical Products Administration (NMPA) has approved pimicotinib (ABSK021) as a treatment for adults with symptomatic tenosynovial giant cell tumors (TGCTs) for which surgery may cause functional limitation or relatively severe morbidity, according to a press release from the developer, Merck KGaA.1

Supporting data for the approval came from the phase 3 MANUEVER trial (NCT05804045), in which investigators assessed the safety and efficacy of pimicotinib among patients with TGCTs who received no prior anti–colony-stimulating factor (CSF) 1 or anti–CSF-1R treatment. At 25 weeks, the objective response rate (ORR) per blinded independent central review (BICR) was 54.0% with pimicotinib vs 3.2% with placebo (P < .0001). Treatment with pimicotinib also correlated with significant improvements in relative range of motion (P = .0003), physical function on the Patient-Reported Outcomes Measurement Information System (PROMIS-PF) scale (P = .0074), worst stiffness (P < .0001), and worst pain (P < .0001).

At a median follow-up of 14.3 months, the ORR had increased from the beginning of the study to 76.2% (95% CI, 63.8%-86.0%) among patients receiving pimicotinib. These updated long-term results were presented at the European Society for Medical Oncology 2025 Congress.

Safety data from the MANUEVER trial showed that pimicotinib was well-tolerated, as the agent showed no signs of cholestatic hepatotoxicity or hair or skin hypopigmentation. In the double-blind treatment phase of the study, 1 patient (1.6%) who received pimicotinib had a treatment-emergent adverse effect (TEAE) leading to treatment discontinuation. Additionally, 7.9% of those in the pimicotinib arm had TEAEs resulting in dose reductions.

“Many people living with TGCT in China have [had] a long and difficult journey due to the lack of approved options beyond surgery, which may not address the needs of patients whose tumors recur or are not amenable to resection,” Professor Niu Xiaohui, director of the Bone and Soft Tissue Tumor Diagnosis and Research Centre at Beijing Jishuitan Hospital, said in the press release.1 “With the approval of pimicotinib based on the results of the global MANEUVER study, health care professionals in China will soon have the opportunity to prescribe their patients an effective and well-tolerated systemic treatment option, offering a much-needed advance in how they manage this challenging condition.”

The international phase 3 MANUEVER trial assessed pimicotinib across 3 parts. In part 1 of the trial, 94 patients were randomly assigned 2:1 to receive pimicotinib at 50 mg once daily (n = 63) or a matched placebo (n = 31) for 24 weeks. Following part 1, patients could proceed to part 2 for 24 additional weeks of treatment. Additionally, those who completed part 2 were eligible for part 3, an open-label extension phase, to undergo extended treatment and safety follow-up.

The trial’s primary end point was ORR at 25 weeks per BICR in the intent-to-treat population based on RECIST v1.1 criteria. Secondary end points included ORR per tumor volume score, relative range of motion, stiffness by Numeric Rating Scale (NRS), pain by Brief Pain Inventory (BPI), and physical function based on PROMIS-PF.

Patients 18 years and older with histologically confirmed, unresectable TGCT and measurable disease per RECIST v1.1 guidelines were eligible for enrollment on the study.2 Other eligibility criteria included having an ECOG performance status of 0 or 1 and adequate organ function and bone marrow function.

In June 2025, China’s Center for Drug Evaluation of the NMPA accepted an application seeking approval for pimicotinib in TGCTs based on data from the MANEUVER trial.3

“This approval is a significant step forward in further strengthening our leadership in rare tumors, while offering patients the opportunity to change the course of their disease and help alleviate symptoms that impact their daily lives. We are now working to make pimicotinib available to patients in China as quickly as possible, as we continue to progress applications with regulatory authorities in additional markets,” Danny Bar-Zohar, chief executive officer of Healthcare and member of the Executive Board of Merck KGaA in Darmstadt, Germany, concluded.1

References

1. Pimicotinib approved as systemic treatment in China for tenosynovial giant cell tumor. News release. Merck KGaA. December 22, 2025. Accessed December 22, 2025. https://tinyurl.com/5adfvsvu
2. Study of pimicotinib (ABSK021) for tenosynovial giant cell tumor (MANEUVER). ClinicalTrials.gov. Updated September 8, 2025. Accessed December 22, 2025. https://tinyurl.com/mru29zsb
3. China’s Center for Drug Evaluation accepts Merck’s application for marketing authorization of pimicotinib for treatment of tenosynovial giant cell tumor. News release. Merck. June 10, 2025. Accessed December 22, 2025. https://tinyurl.com/37k7df8b