Early Study on Drug Shows Great Success in Treating C3 Glomerulopathy

A major first step for a viable treatment option!

Achillion Pharmaceuticals last week reported preliminary proof-of-concept results from its ongoing Phase 2, study of a new treatment for C3 glomerulopathy (C3G)!
C3 glomerulopathy is a group of conditions that cause the kidneys to malfunction. The major features of C3 glomerulopathy include high levels of protein in the urine (proteinuria), blood in the urine (hematuria), reduced amounts of urine, low levels of protein in the blood, and swelling in many areas of the body. Affected individuals may have particularly low levels of a protein called complement component 3 (or C3) in the blood. To read more about this rare disease, click here.

C3 glomerulopathy is very rare, affecting 1 to 2 per million people worldwide.

“Preliminary data from this Phase 2 trial suggest that [the drug in trial] may reverse the AP hyperactivity in C3G based upon the observed improvements in fragment,” commented Milind Deshpande, Ph.D., President and CEO of Achillion. “We are excited by these early data and [its] potential to be a novel and potentially disease-modifying treatment for C3G and IC-MPGN.”

What the study is already showing is that the drug treatment in question, labeled ACH-4471, demonstrated a greater than 50% reduction in proteinuria, over the 14-day treatment period, and demonstrated a favorable tolerability profile.

One of the major features of C3 glomerulopathy includes proteinuria, or high levels of protein in the urine – so these results are very promising!

While this is one of many steps to come that will get us to an FDA approved C3 glomerulopathy treatment, this is nonetheless an important development for the rare condition!

Read more about the study in the Business Insider here.


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