Experimental Treatment for Anemia Associated With Beta Thalassemia Gets Fast Track Designation

According to a story from PR Newswire, the drug developer Protagonist Therapeutics recently announced that its investigational therapy PTG-300 has earned Fast Track designation from the US Food and Drug Administration. PTG-300 is in development for the treatment of chronic anemia, a serious complication that is associated with the rare disease beta thalassemia.

About Beta Thalassemia

Beta thalassemias are a group of heritable disorders affecting the blood. The disorder is characterized by abnormal production of hemoglobin, the substance in red blood cells that is responsible for transporting oxygen. Beta thalassemia is caused by mutations affecting the beta globin gene. Homozygous inheritance of mutated genes results in the most severe form that leads to early death without treatment. Symptoms include anemia, gallstones, and spleen enlargement (which often results in surgical removal). In the most severe type, skeletal deformities and and poor growth also appear. Treatment includes frequent blood transfusions, which can cause complications such as liver and heart problems thanks to excess iron. Treatment is also necessary to prevent iron overload, but it comes with pain and unpleasant side effects. More effective and convenient treatments are needed to improve patients’ quality of life. To learn more about beta thalassemia, click here.

About Fast Track Designation

Fast Track designation is a program from the FDA that can help speed up the development process for products that show the potential to treat serious disease and/or address medical needs that are not being met by currently available medicines. A company whose drug receives Fast Track designation will benefit from closer collaboration with the FDA and more frequent meetings with officials from the agency. There is also the potential for the product to receive accelerated approval. 

PTG-300 previously received Orphan Drug designation from the FDA as a treatment for beta thalassemia. These designations are a reflection of the potential that PTG-300 displays as a new approach for this rare blood disorder. Protagonist plans to kick off as Phase 2 clinical trial of the experimental treatment before the end of 2018. While more research is necessary, the operating mechanism of PTG-300 suggests that it could be useful for a variety of other blood disorders as well, such as myelodysplastic syndromes.

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