Arrowhead's Plozasiran Receives FDA's Prestigious Breakthrough Therapy Designation

Arrowhead Pharmaceuticals has received the FDA’s breakthrough therapy designation for its innovative RNA interference (RNAi) treatment, plozasiran. This designation highlights the drug's promising trial results, particularly in lowering triglycerides and harmful lipoproteins. With this status, Arrowhead can accelerate the development and review timeline for plozasiran, facilitating quicker access to the therapy for patients with serious conditions.

Chris Anzalone, President and CEO of Arrowhead Pharmaceuticals stated, “There are currently no FDA-approved therapies to specifically treat FCS, leaving physicians with very few options to help their patients. Results from clinical studies of investigational plozasiran have been highly encouraging and strongly supportive of further development and commercialisation in multiple patient populations. Receiving FDA breakthrough therapy designation for plozasiran provides important benefits and the potential to expedite the process of getting plozasiran to the patients who need it.”

Plozasiran is designed to complement a healthy diet by lowering triglyceride levels in adults with familial chylomicronaemia syndrome (FCS), a rare genetic condition. This drug also targets apolipoprotein C-III (APOC3), a crucial protein in triglyceride regulation that, when elevated, contributes to higher blood triglyceride levels—a hallmark of FCS.

In clinical trials, plozasiran has demonstrated its ability to decrease triglycerides and other harmful lipoproteins in patients with FCS, severe hypertriglyceridemia (SHTG), and mixed hyperlipidaemia. Looking ahead, Arrowhead Pharmaceuticals plans to file a new drug application for plozasiran with the FDA by the end of the year and, pending a favorable response, will seek approval from other international regulatory bodies.