FDA Grants Breakthrough Therapy Designation to Arrowhead Pharmaceuticals for Plozasiran
Arrowhead Pharmaceuticals has revealed that the U.S. Food and Drug Administration (FDA) has awarded Breakthrough Therapy designation to their investigational drug, plozasiran.
Plozasiran, formerly known as ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapy designed to lower the production of apolipoprotein C-III (APOC3), a protein that contributes to elevated triglyceride levels.
This treatment aims to reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS), a serious genetic condition marked by dangerously high triglyceride levels that can lead to acute pancreatitis. Currently, there are no approved treatments for FCS in the U.S.
The Breakthrough Therapy designation is designed to speed up the development and review of drugs for serious conditions when early evidence suggests significant improvement over existing therapies.
Plozasiran has also received Orphan Drug Designation and Fast Track Designation from the FDA, as well as Orphan Drug Designation from the European Medicines Agency.
Familial chylomicronemia syndrome (FCS) is a severe genetic condition often caused by specific mutations. It leads to triglyceride levels that usually exceed 880 mg/dL, resulting in serious health issues such as acute pancreatitis, chronic abdominal pain, diabetes, fatty liver disease, and cognitive problems. Currently, there are very few treatment options available for FCS.
In clinical studies, plozasiran has shown promise in reducing triglycerides in patients with FCS, severe hypertriglyceridemia (SHTG), and mixed hyperlipidemia. The treatment has a favourable safety profile, and participants from the PALISADE Phase 3 study have been able to continue into a two-part extension phase to receive plozasiran.
