国家药监局批准首个用于腱鞘巨细胞瘤治疗一类创新药

Core Viewpoint - The National Medical Products Administration (NMPA) has approved Bejimai (pimitidine hydrochloride capsules) for the treatment of symptomatic tenosynovial giant cell tumors (TGCT) in adult patients, marking it as the first innovative drug approved for TGCT treatment in China [1][4]. Group 1: Drug Approval and Significance - Bejimai is a colony-stimulating factor-1 receptor inhibitor, providing a new treatment option for TGCT, a rare locally aggressive tumor that can lead to significant joint swelling, stiffness, and functional limitations [1][4]. - The approval is based on the positive results from the global Phase III MANEUVER study, which were presented at the 2025 American Society of Clinical Oncology Annual Meeting [4]. Group 2: Clinical Impact and Patient Needs - Many TGCT patients in China face treatment challenges, as there are limited options beyond surgery, particularly for those with tumor recurrence or inoperable cases [4]. - The approval of Bejimai offers clinicians the opportunity to provide an effective and well-tolerated systemic treatment option, representing progress in the management of this difficult-to-treat disease [4]. - The pain and activity limitations caused by TGCT significantly impact patients' daily lives, leading to considerable mental stress, highlighting the importance of effective treatment options like Bejimai [4].