First regulatory approval in the world for pimicotinib based on positive data from global Phase 3 MANEUVER study
Merck KGaA, Darmstadt, Germany, a global leader in science and technology, announced that the China National Medical Products Administration has approved pimicotinib for adult patients with symptomatic tenosynovial giant cell tumor, or TGCT, for whom surgical removal could lead to functional limitations or significant complications. Pimicotinib, a colony stimulating factor-1 receptor inhibitor developed by Abbisko Therapeutics in Shanghai, is the first Chemical Drug Class 1 approved in China specifically for TGCT treatment.
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Danny Bar-Zohar, CEO Healthcare and member of the Executive Board at Merck KGaA, stated that this first-in-the-world approval reflects the company’s dedication to improving outcomes for patients with rare tumors. He added that the approval strengthens Merck’s leadership in rare tumors while providing patients with a treatment option that can help manage their disease and alleviate symptoms that affect daily life. Bar-Zohar emphasized that the company is now focused on making pimicotinib available in China as soon as possible and pursuing approvals in additional global markets.
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TGCT is a rare tumor that typically affects joints, causing swelling, stiffness, and reduced mobility, which can significantly impair quality of life. Untreated or recurrent TGCT may lead to irreversible damage to bones, joints, and surrounding tissues. The condition has previously been referred to as pigmented villonodular synovitis.
The China NMPA approval was based on data from the global Phase 3 MANEUVER study, where pimicotinib achieved the highest objective response rate reported in a Phase 3 trial of systemic TGCT therapy. At week 25, pimicotinib demonstrated a statistically significant improvement in the primary endpoint of ORR assessed by a blinded independent review committee, with 54 percent of patients responding compared to 3.2 percent with placebo. Pimicotinib also showed significant improvements in secondary endpoints relevant to patients’ daily function, including enhanced joint range of motion, improved physical function on the PROMIS-PF scale, and reductions in worst stiffness and pain. Long-term follow-up presented at ESMO 2025 showed that the ORR increased to 76.2 percent among patients who began pimicotinib treatment at the start of the study.
Prof. Niu Xiaohui, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital, highlighted that patients in China have faced limited treatment options beyond surgery, especially for recurrent or unresectable tumors. She noted that pimicotinib offers healthcare professionals a well-tolerated systemic therapy that represents a meaningful advancement in TGCT management.
Pimicotinib was generally well-tolerated in MANEUVER, with no signs of cholestatic liver toxicity or hair and skin hypopigmentation. During the randomized, double-blind phase, only one patient discontinued treatment due to adverse events, while dose reductions were necessary for 7.9 percent of patients, demonstrating a favorable safety profile. This approval marks a major milestone in providing TGCT patients in China with a first-of-its-kind systemic therapy option.
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