Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ARO-APOC3 for reducing triglycerides in adult patients with familial chylomicronemia syndrome (FCS). ARO-APOC3 was previously granted Orphan Drug designation by the FDA and the European Union.
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. 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.
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 (NDA).
ARO-APOC3 is being investigated in the Phase 3 PALISADE clinical study (NCT05089084) in patients with FCS, the Phase 2 SHASTA-2 clinical study (NCT04720534) in patients with SHTG, and the Phase 2 MUIR clinical study (NCT04998201) in patients with MD.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
LifeSci Advisors, LLC
LifeSci Communications, LLC
Josephine Belluardo, Ph.D.