Avacopan Named Orphan Drug in Europe for Potential to Treat C3 Glomerulopathy

Avacopan Named Orphan Drug in Europe for Potential to Treat C3 Glomerulopathy

Avacopan (CCX168) has been designated an orphan medicinal product by the European Medicines Agency (EMA) for the treatment of a severe kidney disease called C3 glomerulopathy (C3G).

ChemoCentryx said that a multi-center clinical trial of avacopan for the treatment of C3G is set to begin by mid-year.

C3 glomerulopathy refers to a group of rare kidney-related disorders characterized by deposits of proteins from the body’s complement system into and around the glomeruli, the kidney’s filtration units. The disease causes inflammation and disrupts kidney function, with the glomeruli suffering progressive damage. In about half of all patients, C3G leads to renal failure in less than a dozen years, with patients needing to undergo dialysis and kidney transplant.

There is currently no approved therapy for C3G, so patients are usually given non-specific immunosuppressants. The estimated prevalence of C3G is two-to-three per million people.

Avacopan, the lead drug candidate in the ChemoCentryx ‘s orphan and rare disease program, is an oral drug candidate that inhibits the complement C5a receptor, or C5aR, and is being developed for C3G and other inflammatory and autoimmune diseases.

Avacopan blocks the activity of C5a, a component of the complement system and the natural ligand for C5aR. Avacopan was developed to stop the ongoing process of kidney damage in C3G, and aims to stabilize kidney function. In March, the U.S. Food and Drug Administration (FDA) also designated avacopan an orphan drug as a potential treatment for C3G.

“Securing both U.S. and EU orphan drug designations in quick succession for avacopan in the treatment of C3G provides further validation of the drug candidate’s potential,” Thomas J. Schall, PhD, president and chief executive officer of ChemoCentryx said in a press release. “We believe the ability to selectively target the C5a receptor with a patient-friendly small molecule such as avacopan could be a significant breakthrough in the treatment of C3G and other kidney disorders.”

Avacopan is currently being evaluated to treat ANCA-associated vasculitis (AAV), a disease characterized by inflammatory cell infiltration and necrosis of blood vessel walls; and atypical Hemolytic Uremic Syndrome (aHUS), an extremely rare and serious, progressive kidney disease. The FDA has also granted avacopan orphan drug status for its potential in these two diseases, and the EMA for its potential as an AAV treatment.

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