On May 23, 2017, a biopharmaceutical company, ChemoCentryx, Inc., made a huge announcement. Not only has the US Food and Drug Administration (FDA) granted orphan drug designation for their drug, avacopan, which treats patients with C3 glomerulopathy, but now the European Medicines Agency (EMA) has too!
Being granted orphan status is a big deal, and here’s why: Being granted orphan status is basically a huge incentive to continue development, research and marketing on drugs that wouldn’t typically have a huge a ROI to pharma companies. The idea is that since there are things like tax breaks for orphan status drugs, treatment for rare diseases are more likely to be pushed to market.
Long story short, ChemoCentryx has a lot of pressure from now both the US and Europe to make this drug an incredible breakthrough for C3 glomerulopathy patients and research.
Here’s a little more info on the drug: Avacopan is an oral drug that targets the complement C5a receptor. This complementary receptor, C5a, is known to be the driving force behind the destructive inflammatory cells in C3 glomerulopathy and other kidney diseases.
Basically, ChemoCentryx’s avacopan is meant to slow down or stop further kidney damage in C3G patients, which is super exciting because there is no approved treatment for C3G at the moment.
Even more upsetting is that relapse of this disease is common even after kidney transplant. C3 glomerulopathy is actually more like a combination of problems that cause kidney issues. C3G is a degenerative condition, and sadly around half of patients diagnosed with C3G develop end-stage renal disease within 10 years of diagnosis.
End-stage renal disease, ESRD, is a life-threatening condition where the kidneys fail to function and properly filter fluids and wastes. Because these conditions are so scary, and there is no current treatment for C3 glomerulopathy, ChemoCentryx’s new development is highly-anticipated and potentially life-altering.
Maybe this extra push from Europe is all ChemoCentryx needs to make avacopan hit the market as soon as possible, as effectively as possible.