CSPC Pharma's SYH2061 siRNA drug gets US clinical trial approval

CSPC Pharmaceutical Group Limited announced its double-stranded small interfering RNA (siRNA) drug, SYH2061 Injection, has received approval from the U.S. Food and Drug Administration (FDA) to conduct clinical trials in the United States. This Class 1 new chemical drug, independently developed by CSPC, also obtained clinical trial approval from China's National Medical Products Administration in October 2025.

The SYH2061 Injection is an siRNA drug designed for liver-targeted delivery via N-acetylgalactosamine (GalNAc) conjugation. It targets complement component C5 through subcutaneous administration to reduce C5 levels. The drug is distinguished by its ultra-long-lasting gene silencing effect, making it the first independently developed C5-reducing siRNA drug to enter clinical trials in China. It is intended for treating IgA nephropathy and other complement-mediated diseases.

Preclinical studies indicate that SYH2061 demonstrates superior pharmacological activity and extended efficacy compared to similar siRNA products. The drug offers differentiated advantages such as prolonged therapeutic effects, a favorable safety profile, and high patient adherence, highlighting its potential clinical development value.