- Based on the positive global Phase III MANEUVER study, the National Medical Products Administration (NMPA) has granted the world's first regulatory approval for pimicotinib, the first domestically developed systemic therapy for the treatment of Tenosynovial Giant Cell Tumor (TGCT), to our partner Merck KGaA, Darmstadt, Germany, which holds worldwide commercialization rights
- Pimicotinib, a Class 1 innovative drug discovered and developed by Abbisko Therapeutics, received zero-deficiency approval in just over six months following NDA acceptance. During the review, the Center for Food and Drug Inspection (CFDI) conducted its first-ever overseas clinical site inspection for pimicotinib, which it successfully passed. This outcome reflects Abbisko's rigorous R&D standards and regulators' strong recognition of the therapy's clinical value and data quality.
- Pimicotinib's approval for the treatment of TGCT marks a key milestone in the company's advancement from pioneering innovative research to first product commercialization.
SHANGHAI, Dec. 22, 2025 (GLOBE NEWSWIRE) -- Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter, HKEX code: 02256.HK) today announced that the China National Medical Products Administration (NMPA) has approved pimicotinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause functional limitation or relatively severe morbidity. Following this approval, Merck KGaA, Darmstadt, Germany, is working to ensure timely patient access in China.
TGCT is a rare and locally aggressive tumor primarily affecting joints, tendon sheaths, and bursae with considerable morbidity and functional impairment. TGCT is driven by overexpression of colony-stimulating factor-1 (CSF-1) in neoplastic synovial cells which leads to accumulation of colony-stimulating factor-1 receptor (CSF-1R)-expressing inflammatory cells in the tumor. The Chinese expert consensus on clinical diagnosis and treatment of TGCT recommends CSF-1/CSF-1R pathway inhibitors as the preferred systemic treatment for patients with unresectable TGCT (Recommendation Level I; Evidence Level A).
"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," said Prof. Niu Xiaohui, Director of the Bone and Soft Tissue Tumour Diagnosis and Research Centre at Beijing Jishuitan Hospital. "With the approval of pimicotinib based on the results of the global MANEUVER study, healthcare 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."
Pimicotinib is a novel, oral, highly selective, and potent small-molecule CSF-1R inhibitor independently developed by Abbisko Therapeutics. The approval by the China NMPA is based on the positive results from the global Phase III MANEUVER study, in which pimicotinib demonstrated the highest objective response rate (ORR) based on RECIST v1.1 seen in a Phase III clinical trial for the treatment TGCT.
At week 25, pimicotinib demonstrated a statistically significant improvement in the primary endpoint of ORR compared with placebo (54.0% vs. 3.2%, respectively; p<0.0001) as assessed by Blinded Independent Review Committee (BIRC) based on RECIST v1.1. pimicotinib also demonstrated clinically meaningful and statistically significant improvements across secondary endpoints relevant to patient daily life, improving relative Range of Motion (p=0.0003) and physical function as measured by the PROMIS-PF scale (p=0.0074), as well as reducing Worst Stiffness NRS (p<0.0001) and BPI Worst Pain NRS (p<0.0001). Longer-term follow-up data presented at the 2025 European Society for Medical Oncology (ESMO) Congress further demonstrated that pimicotinib provides robust and durable tumor responses for patients with TGCT. With a median follow-up of 14.3 months, ORR per RECIST v1.1 increased from 54% at week 25 to 76.2% (95% CI: 63.8, 86.0), including four patients achieving a Complete Response (CR).
Dr. Yaochang Xu, Founder and Chief Executive Officer of Abbisko Therapeutics, stated: "The approval of pimicotinib is far more than a regulatory milestone-it is a defining moment in Abbisko's journey. It reflects our evolution from pioneers in scientific innovation to a company capable of bringing meaningful therapies to patients. Guided by an unwavering commitment to addressing real clinical needs, we will continue advancing breakthrough, differentiated medicines that have the potential to reshape the future of oncology and other disease areas in China and around the world. As the first domestically independently developed CSF-1R inhibitor approved in China, pimicotinib not only fills a crucial gap for patients with TGCT, but also exemplifies what we can achieve when we pursue bold ideas with global ambition and a relentless focus on impact."
"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," said Danny Bar-Zohar, CEO Healthcare and Member of the Executive Board of Merck KGaA, Darmstadt, Germany. "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."
In the MANEUVER study, pimicotinib also demonstrated a generally well-tolerated safety profile. The median dose intensity remained high at 88.2% during treatment. Most treatment-emergent adverse events (TEAEs) were Grade 1-2. No new safety signals were observed with longer-term follow-up, and there was no evidence of cholestatic hepatotoxicity, drug-induced liver injury, or hair/skin hypopigmentation.
About Pimicotinib
Pimicotinib is a novel, oral, highly selective, and potent small-molecule CSF-1R inhibitor independently developed by Abbisko Therapeutics. Positive results from the global Phase III MANEUVER study of pimicotinib for the treatment of Tenosynovial Giant Cell Tumor (TGCT) were announced in November 2024. Currently, pimicotinib has been approved by the National Medical Products Administration (NMPA) in China for the treatment of adult patients with symptomatic TGCT for which surgical resection will potentially cause functional limitation or relatively severe morbidity. In December 2023, Abbisko entered into an agreement with Merck KGaA, Darmstadt, Germany, pertaining to the commercial rights to pimicotinib, pursuant to which Merck KGaA, Darmstadt, Germany, is responsible for the commercialization of pimicotinib globally.
Outside of China, pimicotinib has been granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) and PRIME Designation by the European Medicines Agency (EMA).
About Abbisko Therapeutics
Founded in April 2016, Abbisko Therapeutics Co., Ltd. (HKEX: 02256.HK), is an oncology-focused biopharmaceutical company based in Shanghai that is dedicated to the discovery and development of innovative medicines to treat unmet medical needs in China and globally. The Company was established by a group of seasoned drug hunters with rich research & development and managerial expertise from top multinational pharmaceutical companies. Since its founding, Abbisko Therapeutics has built an extensive pipeline of innovative programs focused on precision oncology and immuno-oncology.
Please visit www.abbisko.com for more information.
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