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Protagonist Therapeutics blood disorder drug secures US FDA orphan drug status

The U.S. Food and Drug Administration granted an orphan drug designation to Protagonist Therapeutics Inc.'s PTG-300 to treat beta-thalassemia, a blood disorder that reduces the production of hemoglobin — the iron-containing protein in red blood cells that carries oxygen to cells in the body.

David Liu, chief scientific officer and head of research and development of Protagonist, said the disorder usually requires regular and lifelong blood transfusions for survival. "Over time, these transfusions can lead to excessive iron levels in the body that can be toxic and consequently lead to end-stage damage to vital organs such as the liver and the heart."

PTG-300 is an injectable drug that imitates the Hepcidin protein, a key regulator of the entry of iron in blood circulation, in order to help reduce the excessive iron levels.

The company said the phase 1 trial of PTG-300 showed dose-related and sustained reductions in serum iron levels in healthy volunteers. The drug was well-tolerated with no serious side effects or dose-limiting toxicities.

Protagonist intends to begin global clinical trials of PTG-300 for treating beta-thalassemia after its meetings with the U.S. and European regulatory agencies.