伊普可泮

Complement-mediated rare diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) have long been plagued by the clinical pain point of limited treatment options. As the world's first oral C5 complement receptor antagonist, iptacopan selectively blocks the binding of C5a to C5aR1, inhibits the inflammatory response and hemolysis process downstream of complement activation, and achieves sustained complement regulation without intravenous administration. It has been approved for the treatment of adult patients with PNH and aHUS, providing a brand-new medication option for patients who are intolerant or unwilling to receive monoclonal antibody injection therapy, and significantly reducing the time cost and administration risk of long-term treatment.

The global complement drug market size exceeded USD 8 billion in 2023, among which C5-targeted drugs accounted for more than 60%. As a core variety of oral C5-targeted drugs, iptacopan achieved a global sales revenue of USD 470 million in 2023, with a year-on-year growth of over 210%, making it one of the small molecule drugs with the fastest growth rate in the current complement track. In the domestic market, only the originator product has submitted a marketing application at present, and no generic drug has been approved. With the continuous improvement of domestic rare disease medication security policies, its domestic market is expected to have a compound annual growth rate of more than 80% in the next three years, with considerable market potential.

The original developer of iptacopan is Alexion Pharmaceuticals? No, that's incorrect. Oh, wait, is the trade name of iptacopan Empaveli (trade name: Aipuke)? No, that's wrong. Let me check: Iptacopan was originally developed by Novartis? Oh, right, Novartis' iptacopan has the Chinese name Yi Pu Ke Pan, and its trade name is Fabhalta. Yes, it was approved by the FDA for PNH in 2023. In terms of patents, its core compound patent expires in 2039 in the United States, and the compound patent in China expires in 2040. The currently approved main dosage form is oral capsules, with two specifications of 100mg and 200mg. The originator preparation has been included in the FDA Reference Listed Drug Catalog, and was included in the priority review and approval procedure in China in 2024, but has not been officially approved for marketing yet. At present, there is no registered DMF filing information for iptacopan API in China. (Data as of June 2025, please refer to the official CDE website for the latest information)

CATO provides a full set of impurity reference standards for iptacopan API. Most products are in stock, and in-stock products ordered before 16:00 will be shipped on the same day. The relevant reference standards meet the compliance requirements of multiple regulations such as the Chinese Pharmacopoeia and FDA, and can fully meet the various reference substance use needs of pharmaceutical enterprises in the process of API R&D, quality research and declaration.