The United States Food and Drug Administration has awarded Fast Track designation to an investigational drug called PTG-300 for treating chronic anaemia caused by ineffective erythropoiesis in patients who have beta-thalassemia. For more information about this, you can view the source press release here, at PR Newswire.
About Beta-Thalassemia
Thalassemia is a group of inherited conditions that are linked to low levels of haemoglobin, a substance used by red blood cells to transport oxygen around the body. One form of thalassemia is called beta-thalassemia, and the category of beta-thalassemia is further sub-divided into different forms of the condition.
Different forms of beta-thalassemia are associated with different typical symptoms and ages of onset. However, many people with beta-thalassemia may experience an oxygen shortage in parts of the body, and anaemia caused by reduced levels of red blood cells. Anaemia may lead to symptoms such as weakness and fatigue.
About PTG-300
Protagonist Therapeutics is developing a drug that is designed to treat patients who have beta-thalassemia. PTG-300 is a hepcidin mimetic that may be used to replace hepcidin in patients who have low levels of it. Hepcidin is a hormone that is involved in regulating iron absorption, recycling, and use. Iron is important for many processes in the body, but, according to Protagonist’s article, many people with beta-thalassemia have iron levels that are too high and this can cause damage. A hepcidin mimetic like PTG-300 may be able to help regulate levels of iron in the body. So far, PTG-300 has been awarded Orphan Drug Designation for the treatment of beta-thalassemia, and the FDA has also recently granted the drug Fast Track Designation for the treatment of patients with beta-thalassemia who have chronic anaemia due to ineffective erythropoiesis.
Fast Track Designation
The FDA uses Fast Track designation to speed up the development and review process for drugs that may significantly benefit patients who have serious conditions and an unmet medical need. Drugs that are awarded Fast Track status may be eligible for benefits such as more frequent meetings and communication with the FDA, and rolling review (where the FDA reviews parts of an application as they are submitted, rather than waiting until the entire application is submitted). For more information about Fast Track Designation, you can read about it at the FDA’s website by clicking here.
