Noninvasive Testing, Candidate Biomarkers in AATD-Associated Liver Disease

In the absence of a pharmacologic treatment for alpha-1 antitrypsin deficiency (AATD)-associated liver disease, noninvasive testing and biomarkers supporting disease diagnosis and monitoring are of paramount importance.
Data from a pair of phase 2 clinical trials for fazirsiran are helping researchers understand the correlations of noninvasive tests with liver disease burden in patients with AATD-LD and identify biomarkers relevant for therapeutic targeting. An ​​investigational RNA interference therapy, fazirsiran is currently undergoing phase 3 development for AATD-LD and is designed to reduce the production of mutant alpha-1 antitrypsin protein (Z-AAT), subsequently halting the progression of liver disease to allow the liver to regenerate and repair. Fazirsiran was granted Orphan Drug Designation in February 2018 and Breakthrough Therapy Designation in July 2021.
Although liver biopsy is the current gold standard for assessing fibrosis in AATD-LD, inherent issues with its use limit its applicability in clinical practice. Pavel Strnad, MD, full professor and senior physician at University Hospital Rheinisch-Westfälisch Technische Hochschule Aachen in Germany, noted that along with the fact that AATD is a rare disease that doesn’t get much attention, liver biopsy is “out of the question for long-term monitoring.”
Apart from assessing fibrosis, Strnad explained the importance of understanding the process of alpha-1 antitrypsin accumulation in the liver and whether any of the currently used noninvasive features can measure this process. Ultimately, he and a team of researchers determined this was the case, finding that markers known in other disorders also apply to AATD.
Full article: www.hcplive.com/view/pavel-st...
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