Core Viewpoint - The new drug application (NDA) for the oral, highly selective, and effective small molecule CSF-1R inhibitor, Beijiemai (Pimicitin Hydrochloride Capsules), developed by He Yu Pharmaceutical, has been officially accepted by the FDA for the systemic treatment of patients with tenosynovial giant cell tumor (TGCT) [1][2] Group 1: Drug Development and Approval - Beijiemai has been developed by He Yu Pharmaceutical and licensed to Merck for global commercialization [1] - The drug received approval from the National Medical Products Administration (NMPA) in China in December 2025 for symptomatic TGCT adult patients who may experience functional limitations or severe complications from surgical removal [1] - The NDA acceptance by the FDA is based on positive results from the global multicenter, randomized, double-blind, placebo-controlled Phase III MANEUVER study [2] Group 2: Clinical Study Results - The study demonstrated that patients with TGCT treated with Beijiemai showed a significantly higher objective response rate (ORR) compared to the placebo group at week 25, as assessed by the blinded independent review committee (BIRC) using RECIST v1.1 criteria [2] - All secondary endpoints related to key clinical outcomes in TGCT patients showed significant and clinically meaningful improvements, including enhanced joint range of motion and physical function, as well as reductions in stiffness and pain [2] - Long-term data with a median follow-up of 14.3 months indicated a continuous improvement in ORR for patients who started treatment with Beijiemai [2] Group 3: Disease Context and Market Potential - TGCT is a rare locally aggressive tumor occurring in or around joints, leading to swelling, stiffness, and restricted movement, significantly impacting patients' daily activities and quality of life [2] - If untreated or recurrent, TGCT can cause irreversible damage to bones, joints, and surrounding tissues [2] - With the ongoing submissions and future approvals in major global markets, Beijiemai is expected to provide an innovative treatment option that is effective, well-tolerated, and administered once daily, addressing the unmet clinical needs in this disease area [2]
重磅进展！和誉医药腱鞘巨细胞瘤新药贝捷迈 上市申请获FDA受理