Core Viewpoint - Shanghai Heyu Biopharmaceutical Technology Co., Ltd. announced that its new oral, highly selective, and effective small molecule CSF-1R inhibitor, Beijiemai® (Pimitidine Hydrochloride Capsules), has received formal acceptance for New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for the systemic treatment of patients with Tenosynovial Giant Cell Tumor (TGCT) [1][2] Group 1 - Beijiemai® is independently developed by the company and has been authorized to Merck for global commercialization [1][3] - The NDA acceptance is based on positive results from the global multicenter, randomized, double-blind, placebo-controlled Phase III MANEUVER study, which showed a significant improvement in objective response rate (ORR) for TGCT patients treated with Beijiemai® compared to the placebo group [2] - Secondary endpoints related to key clinical outcomes for TGCT patients showed significant and clinically meaningful improvements, including enhanced joint range of motion and physical function, as well as reduced stiffness and pain [2] Group 2 - 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] - Beijiemai® has already been approved by the National Medical Products Administration (NMPA) in China for symptomatic TGCT adult patients who may experience functional limitations or severe complications from surgical resection [3] - The drug has also received Breakthrough Therapy Designation (BTD) from the FDA and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for its overseas applications [3]

Core Viewpoint - The announcement highlights the acceptance of the New Drug Application (NDA) for the novel oral, highly selective, and effective small molecule CSF-1R inhibitor, Beijimai (ABSK021), by the FDA for the treatment of TGCT patients, indicating a significant advancement in addressing an unmet clinical need in this rare tumor type [1][2]. Group 1 - Beijimai is developed by Shanghai Heyu Biopharmaceutical Technology Co., Ltd. and is licensed to Merck for global commercialization [1]. - The NDA acceptance is based on positive efficacy and safety results from the global multi-center, randomized, double-blind, placebo-controlled Phase III MANEUVER study [1]. - The study demonstrated a statistically significant improvement in the objective response rate (ORR) for TGCT patients treated with Beijimai, assessed at week 25 according to RECIST v1.1 criteria [1]. Group 2 - All secondary endpoints related to key clinical outcomes for 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 sustained increase in ORR for patients who received Beijimai treatment from the start of the study [2]. - TGCT is a rare locally aggressive tumor that can lead to joint swelling, stiffness, and limited mobility, significantly impacting patients' daily activities and quality of life [2].