Ionis Pharmaceuticals has recently received the US FDA Fast Track designation for its investigational therapy, olezarsen, in patients with familial chylomicronemia syndrome (FCS). Olezarsen is also being investigated in a Phase III clinical trial for severe hypertriglyceridemia (sHTG) in the US and several EU countries, including France, Germany, Italy, and the UK. If approved, olezarsen has the potential to fulfil an unmet need of more effect triglyceride lipid lowering therapies, says GlobalData, a leading data and analytics company.

Kaya Olczak, Healthcare Analyst at GlobalData, comments: “sHTG often occurs secondary to FCS, but also results from environmental factors including high-fat diets and sedentary lifestyles. In sHTG, triglyceride levels are greatly elevated, increasing the risk of atherosclerotic cardiovascular disease and death. Olezarsen is an antisense RNA oligonucleotide that inhibits the hepatic production of apoCII, thus enhancing clearance and decreasing triglyceride serum levels, and decreasing atherosclerotic risk.”

Waylivra (volanesorsen) is another apoCII inhibitor marketed by Ionis in the EU for patients with FCS with an inadequate response to other TG-lipid lowering therapies, including statins and ezetimibe. The FDA rejected Waylivra approval for FCS in the US in 2018, making this Fast Track designation for olezarsen in FCS a big win for Ionis. This decision from the FDA has pushed the likelihood of approval of olezarsen for sHTG to 72%, according to GlobalData’s Pharma Intelligence Center.

Olczak concludes: “Both Waylivra and olezarsen are being investigated in sHTG, and if Phase III clinical trials prove successful GlobalData predicts Waylivra and olezarsen could launch for sHTG patients as early as 2028 in the US and the EU. This additional approval of Waylivra and olezarsen for sHTG will further bolster Ionis’ dyslipidemia portfolio and strengthen its competitive standing in the dyslipidemia space.”