RARE Daily

Arrowhead Receives FDA Fast Track Designation for ARO-APOC3 as a Treatment for FCS

March 20, 2023

Rare Daly Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Arrowhead Pharmaceuticals’ ARO-APOC3 for reducing triglycerides in adult patients with familial chylomicronemia syndrome (FCS).

Familial chylomicronemia syndrome (FCS) is a rare genetic disorder that causes severely elevated triglyceride levels, which can result in acute and potentially fatal pancreatitis. There are currently no FDA approved therapies to treat FCS.

ARO-APOC3 is Arrowhead’s investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3) being developed as a treatment for patients with severe hypertriglyceridemia (SHTG), mixed dyslipidemia (MD), and FCS. ARO-APOC3 was previously granted Orphan Drug designation by the FDA and the European Union.

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need. The purpose is to get important new drugs to patients earlier. This designation makes Arrowhead eligible for multiple potential benefits including more frequent interactions with FDA, eligibility for accelerated approval and priority review, and rolling review of the new drug application.

ARO-APOC3 is being investigated in the phase 3 PALISADE clinical study in patients with FCS, the phase 2 SHASTA-2 clinical study in patients with SHTG, and the phase 2 MUIR clinical study in patients with MD.



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