China’s Center for Drug Evaluation Accepts MAA for Pimicotinib in TGCT

China’s Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) has accepted the marketing authorization application for pimicotinib (ABSK021) as a class 1 innovative drug for the treatment of adult patients with tenosynovial giant cell tumors (TGCT) who require systemic therapy.¹

The application is supported by data from part 1 of the phase 3 MANEUVER study (NCT05804045), which examined pimicotinib vs placebo in adult patients with unresectable TGCT. During the 2025 ASCO Annual Meeting, investigators presented updated data from MANEUVER, which demonstrated that patients who received the CSF-1R inhibitor (n = 63) achieved an overall response rate (ORR) of 54.0% (95% CI, 40.9%-66.6%) per RECIST 1.1 criteria at week 25 compared with 3.2% (95% CI, 0.1%-16.7%) in the placebo arm (n = 31), representing a difference of 50.7% (95% CI, 37.0%-64.5%; P < .0001).² One patient in the investigational arm achieved a complete response (CR). The median durations of response (DORs) were not reached (NR; range, 0.03+ to 11.76+) and NR (range, not assessed), respectively.

“With the acceptance of our application for pimicotinib and the initiation of the priority review, we aim to offer patients in China the first approved systemic therapy for TGCT, addressing a tremendous unmet need in this country,” Hong Chow, head of China and international, health care business at Merck KGaA in Darmstadt, Germany, stated in a news release.¹ “Pimicotinib has demonstrated the ability to not only shrink tumors that affect [patients’] joints but also improve outcomes like mobility, pain and stiffness, highlighting its potential to be a best-in-class treatment for TGCT. In parallel, we are working to file a new drug application to the US FDA, with additional filings planned in other markets.”

MANEUVER enrolled patients with symptomatic disease due to active TGCT who had measurable disease per RECIST 1.1 criteria with at least 1 lesion measuring at least 2 cm.² Patients were stratified by China vs non-China.

During the double-blinded part 1, eligible patients were randomly assigned 2:1 to receive pimicotinib at a dose of 50 mg daily or placebo. During parts 2 and 3, all patients will receive open-label pimicotinib. Part 1 lasted 24 weeks, part 2 will also last 24 weeks, and part 3 will be an open-label extension.

The primary end point is ORR by blinded independent central review (BICR) per RECIST 1.1 criteria. Key secondary end points include ORR by BICR per tumor volume score (TVS); as well as mean change from baseline in clinical outcomes assessments of range of motion, worst pain numeric rating scale (NRS), worst stiffness NRS, and PROMIS-PF T-score.

Additional findings from MANEUVER part 1 showed that the 25-week ORRs per TVS were 61.9% (95% CI, 48.8%-73.9%) and 3.2% (95% CI, 0.1%-16.7%) in the investigational and placebo arms, respectively. The ORR difference between the 2 groups was 58.7% (95% CI, 45.2%-72.2%; P < .0001). The median DORs were NR (range, 0.03+ to 14.13+) and NR (range, 3.22+ to 3.22+), respectively. One patient in the pimicotinib arm achieved a CR.

In terms of safety, any-grade treatment-emergent adverse effects (TEAEs) occurred in 100% of patients in the pimicotinib arm and 93.5% of patients in the placebo arm by week 25. In the pimicotinib arm, common any-grade clinical TEAEs occurring by week 25 included pruritus (52.4%), face edema (47.6%), and rash (34.9%). Common any-grade clinical TEAEs in the placebo arm included fatigue (22.6%), periorbital edema (9.7%), nausea (6.5%), and headache (6.5%).

In January 2023, the FDA granted breakthrough therapy designation to pimicotinib for patients with TGCT who are not amenable for curative surgery.³ In May 2025, the agent received priority review from the CDE for the treatment of patients with TGCT who require systemic therapy.¹ Pimicotinib has also received breakthrough therapy designation from the NMPA.

References

1. China’s Center for Drug Evaluation accepts Merck KGaA, Darmstadt, Germany’s application for marketing authorization of pimicotinib for treatment of tenosynovial giant cell tumor. News release. Merck KGaA. June 10, 2025. Accessed June 10, 2025. https://www.emdgroup.com/en/news/pimicotinib-china-nda-accept-10-05-2025.html
2. Niu X, Ravi V, Broto JM, et al. Pimicotinib in tenosynovial giant cell tumor (TGCT): efficacy, safety and patient-reported outcomes of phase 3 MANEUVER study. J Clin Oncol. 2025;43(suppl_16):11500. doi:10.1200/JCO.2025.43.16_suppl.11500
3. Abbisko Therapeutics announces that US FDA has granted breakthrough therapy designation for its CSF-1R inhibitor pimicotinib (ABSK021). News release. Abbisko Therapeutics. January 30, 2023. Accessed June 10, 2025. https://www.abbisko.com/newsDetail/127.html