Press Release: Amyndas’ lead candidate AMY-101 receives orphan drug status from the FDA and the EMA for the treatment of C3 glomerulopathy

Amyndas Pharmaceuticals SA recently announced that its lead candidate, the C3 complement inhibitor AMY-101, has been granted orphan designation by the European Medicines Agency (EMA) and the U.S. FDA for the treatment of C3 glomerulopathy (C3G). Specifically, AMY-101 is the first drug to be granted an orphan drug status by the EMA and the U.S. FDA for the indication of C3G. Currently, there is no treatment for C3G. Since the pathology of C3G is due to malfunction of the alternative pathway (AP) of complement, inhibition of complement component C3 is a promising strategy for the development of an effective treatment. Indeed, recent studies have shown that AMY-101 inhibits complement dysregulation in C3G in vitro, presenting a promising therapeutic opportunity and potentially a major advancement for patients with C3G.

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