China’s National Medical Products Administration (NMPA) has approved pimicotinib (ABSK021) as a systemic treatment for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for whom surgical resection is expected to result in functional limitation or relatively severe morbidity.1
Pimicotinib is a colony-stimulating factor 1 receptor (CSF-1R) inhibitor. The approval was supported by data from the global phase 3 MANEUVER study (NCT05804045) that demonstrated among patients treated with pimicotinib an objective response rate (ORR) of 54.0% at week 25 per RECIST 1.1 criteria as assessed by a blinded independent review committee; conversely, this rate was 3.2% in the placebo arm (P < .0001).
In addition to the primary efficacy outcome, pimicotinib was associated with statistically significant and clinically meaningful improvements from baseline across multiple secondary end points, including relative range of motion (ROM; P = .0003) and physical function as measured by the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function scale (P = .0074), as well as reductions from baseline in Worst Stiffness numeric rating scale (NRS; P < .0001) and Brief Pain Inventory Worst Pain NRS (P < .0001). With longer-term follow-up presented at the 2025 ESMO Congress, the ORR per RECIST 1.1 criteria increased to 76.2% (95% CI, 63.8%-86.0%) among patients treated with pimicotinib from study initiation, at a median follow-up of 14.3 months.
“Many people living with TGCT in China have faced 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 Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital in China, stated in a news release. “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.”
How is the MANEUVER study designed?
Pimicotinib NMPA Approval in TGCT: Key Takeaways
- China’s NMPA has approved pimicotinib as a systemic treatment for adults with symptomatic TGCT when surgery may result in functional limitation or morbidity.
- In the MANEUVER trial, pimicotinib significantly improved ORR vs placebo at week 25 (54.0% vs 3.2%), with the ORR in the pimicotinib arm increasing to 76.2% with longer follow-up.
- Pimicotinib demonstrated a generally well-tolerated safety profile, with mostly grade 1 to 2 adverse effects and no new safety signals identified.
MANEUVER is a pivotal, global, 3-part, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of pimicotinib in patients with TGCT who require systemic therapy and have not previously received anti–CSF-1/CSF-1R treatment.2
The primary end point was ORR at week 25 assessed using RECIST 1.1 criteria in the intent-to-treat population. Key secondary end points included ORR by tumor volume score; mean change from baseline to week 25 in ROM of the affected joint, Worst Stiffness NRS, Worst Pain NRS, physical function evaluated using the PROMIS Physical Function scale, and quality of life; and duration of response.
In the double-blind part 1, patients were randomly assigned to receive pimicotinib or placebo. Following completion of part 1, eligible patients were permitted to continue into an open-label part 2 for up to an additional 24 weeks of treatment. Patients who completed part 2 could then enroll in an open-label extension phase (part 3) to allow for extended treatment and longer-term safety follow-up.
What safety profile was observed with pimicotinib in the MANEUVER study?
Pimicotinib demonstrated a generally well-tolerated safety profile.1 The median dose intensity remained high at 88.2% throughout treatment, and most treatment-emergent adverse effects were grade 1 or 2. With longer-term follow-up, no new safety signals were identified, and there was no evidence of cholestatic hepatotoxicity, drug-induced liver injury, or hair or skin hypopigmentation.
“We are continuing to deliver on our commitment to improving the lives of patients with rare tumors with this first-in-the-world regulatory approval of pimicotinib,” Danny Bar-Zohar, chief executive officer of Healthcare and a member of the Executive Board of Merck KGaA, added in the news release. “This approval is a significant step forward in further strengthening our leadership in rare tumors, offering patients the opportunity to change the course of their disease and help alleviate symptoms that [affect] 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.”
References
- Abbisko Therapeutics’ CSF-1R inhibitor pimicotinib approved by the China NMPA, addressing the treatment gap for tenosynovial giant cell tumor. Abbisko Therapeutics. News Release. December 19, 2025. Accessed December 22, 2025. https://www.abbisko.com/newsDetail/229.html
- Study of pimicotinib (ABSK021) for tenosynovial giant cell tumor (MANEUVER). ClinicalTrials.gov. Updated September 8, 2025. Accessed December 22, 2025. https://www.clinicaltrials.gov/study/NCT05804045
