biotech and life sciencesNEW HAVEN: The US Federal Drug Administration FDA has awarded Achillion Pharmaceuticals, Inc. [Nasdaq:ACHN], orphan drug designation for it development drug ACH-4471 for the treatment of patients with C3 Glomerulopathy (C3G).

According to the company, C3G is a “devastating renal disease for which there is no approved therapy.” Adding, “there are estimated to be approximately 4,000 C3G patients in the United States, approximately 4,000 in Europe, and more than 1,000 patients in Japan.

The FDA Orphan Drug Designation program provides incentives for the development of potentially promising drugs to treat, diagnose or prevent orphan diseases and disorders that affect fewer than 200,000 people in the U.S. This designation may provide, under specified conditions, for a seven-year marketing exclusivity period, as well as certain incentives, including federal grants, tax credits and a waiver of PDUFA filing fees.

On November 14, 2017, Achillion announced interim data from the first two patients in an on-going Phase 2 study in C3G patients demonstrated that the current formulation of ACH-4471 achieved “complement alternative pathway inhibition resulting in greater than 50% reduction in proteinuria over the 14-day treatment period and a favorable tolerability profile.”

“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,” commented Milind Deshpande, Ph.D., President and CEO.