Agios Pharmaceuticals Inc. said the U.S. Food and Drug Administration granted a breakthrough therapy designation to Tibsovo to treat certain patients with a type of blood cancer.
The Cambridge, Mass.-based biotech said Tibsovo, or ivosidenib, was given the status to treat patients with relapsed or refractory myelodysplastic syndrome that have a mutation called isocitrate dehydrogenase 1, or IDH1.
Myelodysplastic syndrome, or MSD, is a group of cancers in which immature blood cells in the bone marrow do not mature and therefore do not become healthy blood cells. Symptoms for the disease include tiredness, weakness and shortness of breath due to low levels of red blood cells, frequent and severe infections because of a lesser number of white blood cells, as well as severe nosebleeds and easy bruising due to an inadequate number of platelets.
Agios is currently evaluating the drug in a phase 1 trial for safety, effects on the human body as well as biochemical and physiological impacts in different types of blood cancers including MSD.
The FDA grants breakthrough designations to new medicines that treat serious or life-threatening conditions and are potentially better than existing therapies. The designation expedites the development and review of the drug.
In November, Agios decided to scrap its plan to seek the FDA's approval for Tibsovo to treat a certain type of bile duct cancer following a meeting with the regulator.
Tibsovo is approved in the U.S to treat certain patients with a type of blood cancer known as acute myeloid leukemia.