Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to investigational plozasiran as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a severe and rare genetic disease characterized by extremely high triglyceride levels which can cause acute and potentially fatal pancreatitis. There are currently no approved treatments in the U.S. for FCS.
“There are currently no FDA-approved therapies to specifically treat FCS, leaving physicians with very few options to help their patients,” said Chris Anzalone, Ph.D., President and CEO of Arrowhead. “Results from clinical studies of investigational plozasiran have been highly encouraging and strongly supportive of further development and commercialization 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.”
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies on clinically significant endpoints2. Plozasiran has also previously been granted Orphan Drug Designation and Fast Track Designation by the FDA and Orphan Drug Designation by the European Medicines Agency.
Arrowhead intends to submit a New Drug Application to the FDA by year-end 2024 and plans to seek regulatory approval with additional global regulatory authorities thereafter.
