CDE Accepts Merck’s Pimicotinib NDA for TGCT Treatment
Merck, a global leader in science and technology, has officially announced that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has accepted its application for marketing authorization of pimicotinib. CDE This application pertains to its use as a Class 1 innovative drug intended for adult patients suffering from tenosynovial giant cell tumor (TGCT) who are in need of systemic treatment. This significant development comes after the CDE granted Priority Review status to pimicotinib in May, acknowledging the urgent need for effective treatment options for patients with TGCT and thereby accelerating the review process.
CDE Pimicotinib is an investigational colony-stimulating factor-1 receptor (CSF-1R) inhibitor that has shown considerable promise as a best-in-class therapy. The drug is being developed by Abbisko Therapeutics Co., Ltd. and has already been granted Breakthrough Therapy Designation (BTD) by the NMPA. These designations underline the innovative nature of pimicotinib and its potential to fulfill an unmet clinical need for patients suffering from TGCT.
Hong Chow, Head of China and International at Merck’s Healthcare business, emphasized the importance of this development. “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,” said Chow. She further highlighted pimicotinib’s therapeutic benefits, stating that the drug has demonstrated efficacy in shrinking tumors that impair joint function, CDE while also improving patient outcomes in terms of mobility, pain reduction, and stiffness alleviation. Chow added that Merck is concurrently preparing to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA), with additional applications planned for other international markets.
Tenosynovial giant cell tumor (TGCT) is a rare but debilitating condition that predominantly affects the synovial lining of joints, bursae, and tendon sheaths. Characterized as a locally aggressive and frequently recurring tumor, TGCT is associated with high levels of morbidity. CDE Common symptoms include swelling, joint pain, stiffness, and restricted mobility, all of which can significantly impair the quality of life of affected individuals, many of whom are of working age. In cases where the condition is left untreated or recurs frequently, TGCT can lead to irreversible damage to the bones, joints, and surrounding soft tissues.
CDE Due to the limitations of current treatment options, there is a compelling need for effective and well-tolerated systemic therapies capable of halting tumor growth while also mitigating the clinical symptoms of the disease. Existing therapeutic interventions, such as surgery and radiation, often fall short of delivering long-term relief and can come with a range of complications and risks. As a result, pimicotinib represents a potentially transformative option in the clinical landscape for TGCT.
The CDE’s acceptance of Merck’s application is supported by robust data derived from Part 1 of the global Phase 3 MANEUVER study. This rigorous clinical trial evaluated the efficacy and safety of once-daily oral pimicotinib in patients with TGCT who required systemic treatment. According to the study’s findings, pimicotinib met its primary endpoint by demonstrating a statistically significant improvement in objective response rate (ORR) when compared with placebo. At week 25, the ORR as assessed by a blinded independent review committee (BIRC) was 54.0% for pimicotinib, compared to just 3.2% for the placebo group (p<0.0001).
CDE Beyond the primary endpoint, pimicotinib also showed statistically significant and clinically meaningful improvements across a range of secondary endpoints. These included key patient-reported outcomes related to physical functionality, such as improvements in active range of motion and reductions in stiffness and pain. Such results underscore the drug’s dual ability to inhibit tumor progression and enhance quality of life for patients. The data were presented earlier this month at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, further substantiating pimicotinib’s therapeutic potential.
The MANEUVER study represents one of the most comprehensive global clinical evaluations of a CSF-1R inhibitor for TGCT to date. Its positive results offer strong evidence that pimicotinib could significantly change the treatment paradigm for this rare and challenging condition. The findings from the study have been well-received by the medical community and have sparked considerable interest among oncologists and rheumatologists specializing in rare joint diseases.
The acceptance of this application is not only a milestone for Merck and Abbisko Therapeutics but also a promising step forward for patients in China who have long lacked viable systemic treatment options for TGCT. As the Priority Review process progresses, it is hoped that pimicotinib will soon become the first approved systemic therapy for TGCT in China, thereby addressing a significant unmet medical need and potentially setting a new standard of care.
Looking ahead, Merck has expressed its commitment to pursuing additional regulatory submissions around the globe. The planned NDA filing with the U.S. FDA is a key part of the company’s broader strategy to bring pimicotinib to patients worldwide. Regulatory filings in other regions will follow based on the continued accumulation of clinical data and regulatory feedback.
The development and potential approval of pimicotinib exemplify Merck’s strategic focus on innovation and its dedication to addressing rare and difficult-to-treat conditions. The company’s collaboration with Abbisko Therapeutics also highlights the importance of partnerships in advancing drug development, particularly for diseases with high unmet needs.
In conclusion, the acceptance of Merck’s application for marketing authorization of pimicotinib by the China CDE marks a major milestone in the journey to bring a much-needed treatment option to TGCT patients. With compelling clinical data, regulatory designations that support expedited review, and a strategic roadmap for global submissions, pimicotinib is poised to become a groundbreaking therapy that not only addresses tumor progression but also significantly improves patient outcomes in terms of quality of life and functional ability.
As Merck continues to advance this candidate through the regulatory landscape, the medical community and patient advocacy groups are closely watching its progress. Should it gain final approval, pimicotinib could represent a new chapter in the management of TGCT, bringing hope to thousands of patients who currently face limited and often unsatisfactory treatment options.
