Protagonist Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTG-300, the company’s sub-cutaneous injectable hepcidin mimetic for the treatment of beta-thalassemia.

“Beta-thalassemia is a rare genetic blood disorder that is characterized by impaired red blood cell production that can result in life-threatening chronic anemia, usually requiring regular and life-long blood transfusions for survival. Over time, these transfusions can lead to excessive iron levels in the body which can be toxic and consequently lead to end-stage damage to vital organs such as the liver and the heart,” said David Y. Liu, Ph.D., chief scientific officer and head of Research and Development of Protagonist Therapeutics. “As a hepcidin mimetic, PTG-300 is designed to help reduce these excessive iron levels and thereby it may lead to improvements in anemia and decreased need for blood transfusions and chelation therapy.”

Protagonist recently completed a Phase 1 study of PTG-300 that established pharmacodynamic-based clinical proof-of-concept by achieving dose-related and sustained reductions in serum iron levels in normal healthy volunteers. It was well tolerated with no serious adverse events or dose-limiting toxicities. The company intends to initiate a global clinical trial with PTG-300 in patients with beta-thalassemia following our upcoming meetings with the U.S. and European regulatory agencies.