The Food and Drug Administration (FDA) has granted Fast Track designation to Protagonist Therapeutics’ PTG-300 for the treatment of chronic anemia due to ineffective erythropoiesis in patients with beta-thalassemia. Previously, this injectable hepcidin mimetic was granted Orphan Drug status by the FDA for this indication. 

In patients with beta-thalassemia, abnormally low hepcidin levels can result in the body absorbing and storing more iron than is needed, leading to iron overload. Treatment with a hepcidin mimetic may help restore iron homeostasis in these patients.

A Phase 1 trial in healthy volunteers established a pharmacodynamic proof-of-concept based on dose-related reduction in serum iron levels. The Company is currently planning a Phase 2 trial of PTG-300 in beta-thalassemia patients which they expect to begin before the end of the year.

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“The Phase 2 trial incorporates an open label trial design with objective endpoints that will enable us to assess the performance of PTG-300 in an accurate and timely manner,” said Dinesh V. Patel, PhD, Protagonist President and CEO. 

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