Core Viewpoint - Shanghai Heyu Biopharmaceutical Technology Co., Ltd. announced that its self-developed oral and highly selective small molecule CSF-1R inhibitor, Beijiemai (pimitinib hydrochloride capsules), has received formal acceptance of its New Drug Application (NDA) by the U.S. Food and Drug Administration (FDA) for the systemic treatment of patients with tenosynovial giant cell tumors (TGCT) [1][2]. Group 1 - Beijiemai is a novel, oral, highly selective, and efficient small molecule CSF-1R inhibitor developed independently by the company [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]. - 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 reduced stiffness and pain [2]. Group 2 - TGCT is a rare locally aggressive tumor that occurs 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 company has reached an agreement with Merck for the global commercialization rights of Beijiemai, which has also received breakthrough therapy designation (BTD) from the FDA and priority medicine (PRIME) designation from the European Medicines Agency (EMA) [3].
和誉医药腱鞘巨细胞瘤新药贝捷迈 上市申请获FDA受理