The Food and Drug Administration (FDA) has granted Orphan Drug Designation to Livantra (trimetazidine; Martin Pharmaceuticals) for the treatment of acute-on-chronic liver failure (ACLF).
Livantra is a repurposed, reformulated version of trimetazidine, a former cardiovascular therapy used in Europe. It is thought to work by shifting the energy generation in the liver from fatty acid oxidation to glucose oxidation so that less toxic molecules are produced. This results in less damage to hepatocytes and thus fewer hepatocyte death. Livantra is also thought to help preserve cellular energy, leading to higher ATP levels in hepatocytes.
Patients with ACLF have acute decompensation of cirrhosis associated with the presence of 1 or more organ failure. Currently, there is no specific treatment for ACLF. Rather, treatment is based on organ support and treatment of associated complications. Livantra may help rate of survival without transplantation or help improve survival until patients can receive a new liver.
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