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.
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. PTG-300 therapy may also be potentially beneficial in other diseases such as myelodysplastic syndrome (MDS), hereditary hemochromatosis (HH), polycythemia vera (PCV), siderophilic infections, and liver fibrosis which provide additional opportunities for future development. 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 and causes organ damage over time. Abnormally low hepcidin levels, caused by genetic mutations or secondary pathology, can result in the body absorbing and storing more iron than is needed, leading to iron overload.
The FDA grants Orphan Drug Designation to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the United States. The designation allows the sponsor of the drug to be eligible for a seven-year period of U.S. marketing exclusivity on approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.