Rare Kidney Disease Treatment Gets FDA’s Orphan Drug Designation

Last month, the FDA has granted Achillion Pharmaceuticals orphan drug designation to ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G).

According to the FDA, the Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S.

The purpose of this program is to provide incentives for the development of treatment drugs for very rare, underserved conditions – like 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.

You can read more about C3G here, and Patient Worthy’s previous coverage of Achillion’s C3G treatment efforts, click here.

“We are pleased that the FDA has granted orphan drug designation to ACH-4471 for treatment of C3G, and we look forward to additional data from the ongoing extended release formulation study. We are keenly aware of the unmet need for patients and we are committed to advancing ACH-4471 for C3G as we believe we have an opportunity to develop a potentially disease-modifying therapy, based on the unique mechanism of action, for ACH-4471,” said Milind Deshpande, Ph.D., President and CEO. 

The next step is the initiation of bioavailability study of extended release formulations of ACH-4471, which CEO Deshpande referenced in the above statement. These formulations are extended release oral tablets, which Achillion hopes will achieve once or twice daily dosing in patients.

Onward and upward! Great news for those in the C3G community.

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