CSPC drug receives China trial approval for rare disease

CSPC Pharmaceutical Group announced that its high-concentration hydroxocobalamin hydrochloride injection has secured clinical trial approval from China's National Medical Products Administration. The trials will focus on treating methylmalonic acidemia (MMA) in Chinese patients. MMA, a rare autosomal recessive disorder, lacks a domestically or internationally approved hydroxocobalamin injection targeting the indication, creating a significant unmet medical need. This clinical approval is for methylmalonic acidemia with elevated homocysteine level (cbl C type) in children. CSPC's product becomes the first hydroxocobalamin injection approved in China for clinical trials in MMA. The company believes this development provides promising clinical value and a potential new treatment option for patients.