Local Company’s Drug Treatment Earns FDA Fast Track Designation

Pasadena-based Arrowhead Pharmaceuticals Inc. announced Monday it has received Fast Track designation from the US Food and Drug Administration (FDA) for ARO-APOC3, a new treatment being developed to reduce triglycerides in adult patients with a rare genetic disorder called familial chylomicronemia syndrome (FCS). 

The disorder causes dangerously high levels of triglycerides in the blood, which can lead to potentially fatal pancreatitis.

ARO-APOC3 is a RNAi therapeutic that targets apolipoprotein C-III (APOC3), and is also being investigated as a treatment for severe hypertriglyceridemia and mixed dyslipidemia. 

The FDA’s Fast Track process is designed to speed up the review and development of drugs to treat serious conditions, and provides several benefits to Arrowhead, including more frequent interactions with the FDA and eligibility for accelerated approval and priority review. 

ARO-APOC3 was previously granted Orphan Drug designation by both the FDA and the European Union, and is currently being investigated in three clinical trials.

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.