Arrowhead Pharmaceuticals receives breakthrough therapy designation from the FDA.
Arrowhead Pharmaceuticals has been granted breakthrough therapy designation from the US Food and Drug Administration (FDA) regarding its potential RNA interference (RNAi) therapeutic, plozasiran. The treatment is said to have shown “promising results” in trials by reducing triglycerides and other atherogenic lipoproteins. By obtaining this designation, Arrowhead Pharmaceuticals can speed up its development and review processes of the drug for serious conditions.
Chris Anzalone, President and CEO of Arrowhead Pharmaceuticals said: “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 intended to be used with a healthy diet to cut down triglycerides in adults with the rare genetic disease, familial chylomicronaemia syndrome (FCS). The drug can also reduce the production of apolipoprotein C-III (APOC3), which is a key regulator of triglyceride metabolism. Additionally, high levels of APOC3 can increase triglyceride levels in the blood, which is a characteristic of FCS.
So far in clinical studies, plozasiran has been seen to reduce triglycerides and other atherogenic lipoproteins in patients with FCS, severe hypertriglyceridemia (SHTG) and mixed hyperlipidaemia.
Looking into the future, Arrowhead Pharmaceuticals is aiming to submit a new drug application for plozasiran to the FDA by the end of the year. If the company receives positive feedback, it plans to seek regulatory approval from additional global authorities.