molecules of the month

In 2022, Amgen acquired ChemoCentryx for $4B in cash, primarily for avacopan, a first-in-class treatment approved in 2021 for a rare automimmune condition, anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV). Scientifically, it is notable that avacopan is the first small molecule targeting the complement pathway to be approved after decades of research since the early ‘90s, attempting to leverage understanding of the complement system to treat immunological conditions. The molecule blocks the C5aR GPCR, a receptor for the inflammatory complement component, C5a.  Standard treatment for AAV includes cyclophosphamide, a mustard agent most well-known for its use in chemo, highlighting the unmet medical need in the indication. Avacopan is much better tolerated, acting specifically through the GPCR target, C5aR, the receptor for…