Abbisko Therapeutics Achieves Milestone with NMPA Acceptance for Pimicotinib Targeting TGCT Treatment

Abbisko Therapeutics Achieves a Significant Milestone in Oncology

Abbisko Therapeutics Co., Ltd. has achieved a noteworthy milestone for its innovative treatment, Pimicotinib, as the China National Medical Products Administration (NMPA) has officially accepted the New Drug Application (NDA). This marks a pivotal moment not only for the company but also for the medical community battling Tenosynovial Giant Cell Tumor (TGCT).

Pimicotinib is a highly selective small-molecule CSF-1R inhibitor that shows considerable promise in addressing the unmet medical needs of adult patients with TGCT. This disease primarily affects the joints, causing symptoms such as pain, swelling, and stiffness, significantly impairing patients' daily activities, especially those in their working years. The acceptance of this application underlines the potential for a groundbreaking treatment that could drastically improve the quality of life for those affected by this condition.

Professor Niu Xiaohui, who leads the Bone and Soft Tissue Tumor Diagnosis and Research Centre at Beijing Jishuitan Hospital, expressed optimism regarding Pimicotinib's potential. According to him, the results from the recent MANEUVER study present strong case data on how Pimicotinib could redefine treatment protocols for TGCT as it manages to not only shrink tumors but also enhance mobility and alleviate pain.

Clinical Trial Insights

The NDA submission follows a series of robust clinical trials, specifically the PART 1 of the Phase 3 MANEUVER study. In this study, patients receiving Pimicotinib showed a statistically significant improvement in the objective response rate (ORR), marking 54.0% compared to just 3.2% in the placebo group—a result deemed highly significant (p<0.0001). Furthermore, additional patient-reported outcomes indicated notable advancements in physical function, active range of motion, and a reduction in symptoms like pain and stiffness, all crucial for enhancing the patient experience and functional capacity.

The findings from this study were prominently presented at the recent 2025 ASCO Annual Meeting, underscoring its significance in the oncology research space. Beyond China, Pimicotinib is already receiving global attention, having garnered Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and PRIME Designation from the European Medicines Agency (EMA).

Partnership with Merck

Further strengthening its market position, Abbisko recently forged a partnership with Merck, allowing the latter to handle the commercialization rights for Pimicotinib at a global level. This collaboration is expected to accelerate the drug’s availability to patients in need, positioning it as a leading treatment in the market.

Yao-Chang Xu, Chairman and CEO of Abbisko, highlighted this regulatory acceptance as not just a corporate milestone but a reflection of their commitment to innovative drug development. He expressed optimism about Pimicotinib, which exemplifies a meaningful leap forward in oncology, emphasizing its unique attributes that cater to the specific needs of TGCT patients.

Future Perspectives

With the NMPA's acceptance and subsequent priority review status granted to Pimicotinib, Abbisko aims to fill a crucial gap in the treatment landscape for TGCT in China, where options remain limited. The focus now shifts towards navigating the approval process efficiently to introduce this novel therapy as the first systemic treatment available in the country for TGCT patients—signifying hope for many.

As Abbisko continues its push towards making Pimicotinib available, parallel efforts are underway to file an NDA with the U.S. FDA while exploring additional international markets for expanded access. This reflects the company’s dedication to not only addressing local medical needs but also making an impact on a global scale.

Abbisko Therapeutics, founded in 2016, is an oncology-centric biopharmaceutical company based in Shanghai. The company has committed itself to developing innovative solutions to tackle critical healthcare challenges faced by patients globally. Through this application and its extensive pipeline of programs focused on precision oncology and immuno-oncology, Abbisko is poised to make significant strides in transforming patient outcomes in the field of cancer treatment.