In a nationwide, multi-center clinical trial, scientists identified a promising new drug medication that improved liver fibrosis in NASH patients by 27%.
Scientists from the University of California San Diego School of Medicine have led an investigation into a promising treatment for individuals with NASH-related fibrosis.
The findings, recently published in The New England Journal of Medicine, indicate that a drug that mimics a hormone in the body improved both liver fibrosis, or scarring of the liver, and liver inflammation in patients with NASH.
“Identifying an effective drug for NASH is extremely promising for patients as currently there are no FDA-approved therapies for this condition,” said Rohit Loomba, MD, the study’s first author and chief of the Division of Gastroenterology and Hepatology at UC San Diego School of Medicine. “NASH can adversely impact the quality of life in patients and can progress to cirrhosis. Its complications can lead to death or liver transplantation.”
“Our findings will further the science of this disease and provide a potential new treatment option to those affected by NASH-related fibrosis.”
The researchers found that the drug, called Pegozafermin, mimicked fibroblast growth factor 21 (FGF21) — a liver-secreted peptide hormone that is naturally produced in the body.
FGF21 controls energy use in the body and lipid metabolism in the liver. It has also been shown in previous studies to lower blood glucose and
Of the patients who received the drug at a higher dose, approximately 27% showed an improvement in liver fibrosis, relative to 7% of the patients who received the placebo. The most frequently reported side effect from the drug was gastrointestinal in nature, including nausea.
Currently, there are no medications approved by the U.S. Food and Drug Administration available for the treatment of NASH, which is a type of nonalcoholic fatty liver disease (NAFLD).
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Loomba adds next steps for this research will be a larger, multi-center, international trial with a more diverse patient population and a longer treatment period to better assess the safety of the drug.
“If successfully shown to be both safe and effective in a larger Phase 3 trial, this drug could be used to treat millions of patients with NASH, including our patients at UC San Diego Health,” said Loomba.
Reference: “Randomized, Controlled Trial of the FGF21 Analogue Pegozafermin in NASH” by Rohit Loomba, M.D., M.H.Sc., Arun J. Sanyal, M.D., Kris V. Kowdley, M.D., Deepak L. Bhatt, M.D., M.P.H., Naim Alkhouri, M.D., Juan P. Frias, M.D., Pierre Bedossa, M.D., Ph.D., Stephen A. Harrison, M.D., Donald Lazas, M.D., Robert Barish, M.D., Mildred D. Gottwald, Pharm.D., Shibao Feng, Ph.D., Germaine D. Agollah, Ph.D., Cynthia L. Hartsfield, Ph.D., Hank Mansbach, M.D., Maya Margalit, M.D. and Manal F. Abdelmalek, M.D., M.P.H., 24 June 2023, New England Journal of Medicine.
DOI: 10.1056/NEJMoa2304286
The study was funded by 89bio, ENLIVEN, and the National Institute of Diabetes and Digestive and Kidney Diseases.