Posted by Michael Wonder on 27 Sep 2018
Protagonist Therapeutics announces fast track designation granted by U.S. FDA to hepcidin mimetic PTG-300
27 September 2018 - Designation applies to development for the treatment of chronic anemia in patients with beta-thalassemia.
Protagonist Therapeutics today announced that the U.S. FDA has granted fast track designation to therapeutic candidate PTG-300 for the treatment of chronic anaemia 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 characterised 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," commented 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."
