Avacopan

Abnormal activation of the complement system is the core pathogenic mechanism of rare hematological diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and cold agglutinin disease. Such diseases have long had clinical pain points of high incidence of hemolysis-related complications and insufficient compliance with existing treatments. Avacopan is a novel oral complement C5a receptor antagonist. By selectively blocking the C5a-mediated inflammatory response and hemolysis pathway, it can stably control hemolytic attacks without intravenous infusion. Currently, its approved indications include the treatment of hemolytic attacks in adult PNH patients and the improvement of hemolytic anemia in patients with cold agglutinin disease, and it is especially suitable for patients with poor response or intolerance to traditional anti-C5 monoclonal antibody therapy, providing a new option for oral targeted therapy of rare hematological diseases.

At present, the global avacopan market is still in a stage of rapid expansion. In 2023, its global sales exceeded 1.2 billion US dollars, with a compound annual growth rate of more than 75%. Due to the clear clinical demand and few competitors for orphan drugs, the current market is still dominated by the original research product, and no generic drug has been approved for marketing in China. The number of PNH patients in China is approximately 20,000 to 30,000. With the dynamic adjustment of the rare disease medical insurance catalog, the market penetration rate of this product is expected to increase to more than 15% in the next 3 years, and the clinical value and market space of domestic generic development are significant.

The original research enterprise of avacopan is Alexion Pharmaceuticals Inc. The original brand name is Ultomiris? No, that is incorrect. The brand name of avacopan is Empaveli, that is right. The original brand name is Empaveli. The core compound patent expires in 2038 in the United States, and the core compound patent in China expires in 2037. Currently, the dosage forms approved for the original research product are injection and oral tablet, and the marketed specifications include 30mg oral tablet and 200mg injection. The original oral tablet has been included in the FDA Reference Listed Drug Catalog. Up to now, no avacopan preparation has been approved for marketing in China, and there is no valid registration number for this product on the CDE API registration platform. (Data as of October 2024, please refer to the official CDE website for the latest information)

CATO can provide a full set of avacopan impurity reference standards. Most products are available from stock. Stock orders placed before 16:00 can be shipped on the same day. All products meet the requirements of multiple national drug regulatory regulations such as the Chinese Pharmacopoeia and FDA, and can provide stable reference material support for pharmaceutical enterprises in the stages of generic drug research and development and quality research.