Protagonist Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to therapeutic candidate PTG-300 for the treatment of chronic anemia due to ineffective erythropoiesis in patients with beta-thalassemia. PTG-300 is an injectable hepcidin mimetic that has been granted Orphan Drug Designation by the FDA for beta-thalassemia, a rare disease characterized by iron overload.
"We are pleased to have received Fast Track designation for PTG-300 as recognition of its potential in beta-thalassemia, our initial area of focus and a rare disease with unmet medical need," said Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "We remain on track to initiate a Phase 2 clinical trial in beta-thalassemia patients by the end of the year. 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. In addition, the therapeutic mechanism of PTG-300 suggests potential for future development in the treatment of a broad range of blood disorders, including hereditary hemochromatosis and rare diseases such as polycythemia vera and myelodysplastic syndrome."
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The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and that demonstrate the potential to address unmet medical needs. Drugs that receive this designation benefit from more frequent interactions and meetings with the FDA and potential pathways for expedited approval.
PTG-300, an injectable hepcidin mimetic, is currently in clinical development for the potential treatment of beta-thalassemia, a rare disease characterized by chronic anemia and iron overload. Hepcidin is a natural peptide hormone that is the main regulatory hormone governing iron absorption, recycling and utilization by the body. Iron plays an essential role in various body functions, especially blood formation, but too much iron is toxic, resulting in tissue and organ damage over time. Abnormally low hepcidin levels, caused by genetic mutations or secondary pathology, can be replaced by a hepcidin mimetic to restore iron homeostasis. PTG-300 has been granted Orphan Drug Designation by the FDA for development in the treatment of beta-thalassemia. Treatment of patients with myelodysplastic syndromes, hereditary hemochromatosis and polycythemia vera represent additional opportunities for future development of PTG-300.