In September 2023, Ionis Pharmaceuticals unveiled positive Phase III clinical data for olezarsen, a ligand-conjugated antisense (LICA) designed to inhibit apolipoprotein C-III (apoC-III). Phase III studies showed a significant reduction in triglycerides in patients with familial chylomicronemia syndrome (FCS) taking 80mg of olezarsen monthly for six months. The drug holds the potential to overcome dyslipidemia unmet needs and is expected to reach $849 million in sales by 2032, according to GlobalData, a leading data and analytics company.

Dr Shireen Mohammad, Cardiovascular & Metabolic Disorders Analyst at GlobalData, comments: “According to key opinion leaders (KOLs) interviewed by GlobalData, there is an unmet need for drugs that treat severe genetic disorders, including FCS and familial hypercholesterolemia (FH). Currently, there are no FDA-approved therapies to treat FCS. Therefore, there is a critical unmet need for new treatments for FCS to effectively lower triglyceride levels and prevent the complications associated with this disorder.

A KOL interviewed by GlobalData also noted: “Olezarsen is a wonderful drug for FCS. The good news is olezarsen cures FCS. It does. The problem is that in the entire US, there are 100 people with FCS. That’s the bad news. But there is good news. And that is this, olezarsen is also being developed for severe hypertriglyceridemia and that, instead of being 0.0001% of the population is actually 2% of the population. So, 2% of the entire US population will be eligible for olezarsen. So that is the good news. The bad news is if they were selling to FCS patients only, they would charge half a million dollars per patient per year.”

Hence, GlobalData believes that olezarsen will have an impact on the dyslipidemia market.

Dr Mohammad concludes: “If approved, olezarsen would be the first available treatment for FCS, a rare genetic disease that can lead to pancreatitis attacks. However, due to the lack of treatments for patients with these rare diseases, this therapy is expected to come at a significant cost, but reimbursement is almost guaranteed for patients eligible for treatment.”