RNAi Therapy for Homozygous Familial Hypercholesterolemia Gets Orphan Drug Designation

The Food and Drug Administration (FDA) has granted Orphan Drug designation to ARO-ANG3 (Arrowhead Pharmaceuticals) for the treatment of homozygous familial hypercholesterolemia.

ARO-ANG3 is a subcutaneously administered ribonucleic acid interference (RNAi) investigative treatment that works by reducing production of angiopoietin-like protein 3 (ANGPTL3), an inhibitor of lipoprotein lipase and endothelial lipase in the liver. The inhibition of ANGPTL3 has been shown to lower serum LDL as well as serum and liver triglyceride. 

The Orphan Drug designation is based on an ongoing phase 1 study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of ARO-ANG3 in 70 healthy adult volunteers and dyslipidemic patients. The primary end point of the study is the number of patients with adverse events possibly or probably related to treatment up to 113 days post-dose.

The study has an estimated completion date of December 30, 2019.

For more information visit arrowheadpharma.com.

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