After about 30 years of research, St. Louis University’s Jeffrey Teckman, MD, professor of biochemistry and molecular biology, and his colleagues have a possible new treatment for a genetic liver disease. Alpha-1 antitrypsin deficiency is an inherited disorder that may cause lung disease and liver disease.
The breakthrough drug developed by the team of researchers and pharmaceutical companies experienced tremendous success in a small clinical and is now undergoing a larger trial before potential approval by the U.S. Food and Drug Administration.
The injection of fazirsiran could be one of the first treatments for people with liver disease caused by the condition. Currently, the only approved treatment is a liver transplant.
A key finding in the first clinical trial was that the drug allowed patients’ livers to heal, in addition to reducing the build up of the deformed protein causing damage.