The FDA has granted breakthrough therapy designation to Arrowhead Pharmaceuticals’ plozasiran, coupled with diet, to decrease triglycerides in patients with familial chylomicronemia syndrome, according to a company release.
A rare genetic hyperlipidemia, familial chylomicronemia syndrome (FCS) is characterized by extremely elevated triglyceride levels, which can increase risk for acute and potentially fatal pancreatitis, abdominal pain, diabetes and hepatic steatosis. A first-in-class investigational RNA interference therapeutic, plozasiran is intended to inhibit the production of apolipoprotein C-III, which will in turn reduce triglycerides and return lipids to normal levels.
However, with this breakthrough therapy status, plozasiran is entering a tight race to market against Ionis Pharmaceuticals and its rival therapeutic, olezarsen,which attained orphan drug status for the treatment of FCS earlier this year.
“There are currently no FDA-approved therapies to specifically treat FCS, leaving physicians with very few options to help their patients,” Chris Anzalone, PhD, president and CEO of
Arrowhead, said in a press release. “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.”
The agency based its decision on phase 3 data from the PALISADE trial, which assessed the efficacy and safety of plozasiran compared with placebo in adult patients with FCS. The primary outcome was the percent change from baseline to 10 months0.
According to the release, results showed that plozasiran lowered triglyceride levels by 80% from baseline and reduced the risk of patients with FCS developing acute pancreatitis by 83%.
The company noted that it intends to submit a new drug application to the FDA by the end of the year, then pursue submissions with other global regulatory authorities.
The FDA grants breakthrough designation to expedite development of drugs or devices intended to treat serious conditions and supported by clinical evidence indicating that the product may demonstrate substantial improvement over currently available drugs or devices.
Breakthrough designation is intended to accelerate drug/device development, assessment and review for premarket approval, 510(k) clearance and de novo marketing authorization, while still meeting FDA standards for safety and effectiveness.
