China’s drug regulator has approved pimicotinib, delivering the first systemic treatment option for adults with symptomatic tenosynovial giant cell tumor (TGCT) where surgery could cause serious functional impairment.

 

Global pharma giant Merck, Germany, said the National Medical Products Administration (NMPA) granted approval following Priority Review, making pimicotinib the first Chemical Drug Class 1 therapy approved in China for TGCT. The drug, a colony stimulating factor-1 receptor (CSF-1R) inhibitor, was developed by Abbisko Therapeutics Company of Shanghai.

 

“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.”

 

TGCT is a rare but locally aggressive joint tumor that causes swelling, stiffness and loss of mobility, often affecting otherwise healthy adults. Without effective treatment, or in recurrent disease, TGCT can permanently damage joints, bone and surrounding tissue. The condition has historically been referred to by multiple names, including pigmented villonodular synovitis (PVNS).

 

The approval is based on results from the global Phase 3 MANEUVER study, where pimicotinib delivered the highest objective response rate (ORR) reported in a Phase 3 systemic TGCT trial. 

 

Patients also experienced statistically significant and clinically meaningful improvements in outcomes tied to daily functioning, including range of motion, physical function and reductions in stiffness and pain. 

 

“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.”

 

In the MANEUVER trial, pimicotinib was generally well tolerated, with no evidence of cholestatic liver toxicity or hair and skin hypopigmentation. During the randomized, double-blind phase, treatment discontinuation due to adverse events occurred in one patient (1.6%), while dose reductions were required in 7.9% of patients receiving the drug.

 

The approval positions pimicotinib as a potential new standard of care for TGCT in China and signals growing momentum for systemic treatments in rare orthopedic tumors.