全球首款儿童软骨发育不全治疗药物在国内申报上市：上半年涨了7倍的舒泰神，股价一周下跌23% | 掘金创新药

Market Performance - The pharmaceutical and biotechnology index increased by 2.88% from September 1 to September 5, while the innovative drug index rose by 1.23% during the same period [3] - The Hang Seng Healthcare Index saw a significant increase of 7.07% in the same timeframe [3] Clinical Trials - A total of 48 clinical trial registration information was disclosed by the National Medical Products Administration from September 1 to September 5, with 21 trials in Phase II or above covering various fields such as oncology, dermatology, immunology, and digestion [4] New Drug Approvals - The first global innovative drug for delaying the progression of Type 1 diabetes was approved in China, developed by Sanofi [7] - The injection of Tzield (替利珠单抗) was approved for use in children aged 8 and above and adults with Stage 2 Type 1 diabetes to delay progression to Stage 3 [8] Rare Disease Treatments - The application for the injection of Vosoritide, developed by BioMarin Pharmaceutical for treating achondroplasia in children, has been accepted for review in China [10] - Achondroplasia is a rare skeletal development disorder with a global incidence of approximately 3.72 to 4.60 per 100,000, and it is included in China's rare disease directory [10] - Vosoritide is the first drug targeting the underlying cause of achondroplasia, with traditional treatments only alleviating symptoms [10] Company Insights - Shuyuan Pharmaceutical has seen a remarkable increase of over 700% in stock price over the past six months, driven by the development of a new drug for hemophilia [12][13] - The drug STSP-0601, currently in the approval stage, has shown a 12-hour effective hemostatic rate of 81.94% in Phase IIb trials for hemophilia patients with inhibitors, indicating its potential effectiveness [16][17] - The global market for hemophilia with inhibitors is estimated at approximately 27.7 billion yuan, with potential domestic sales exceeding 2 billion yuan [17] Industry Trends - The Chinese biopharmaceutical sector has rapidly advanced, with the country now being a leader in innovative drug development, accounting for 38% of global large pharmaceutical transactions in early 2025 [19][20] - The trend of Chinese innovative drugs entering global markets is expected to continue for another 12 to 18 months, emphasizing the importance of product quality and robust clinical data [20]