In September 2023, Ionis unveiled positive Phase III clinical data for olezarsen, a ligand-conjugated antisense 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 per month for six months. The drug holds the potential to overcome unmet dyslipidemia needs and is expected to reach $849m in sales by 2032, according to GlobalData.

According to key opinion leaders interviewed by GlobalData, there is an unmet need for drugs that treat severe genetic disorders, including FCS and familial hypercholesterolemia. Currently, there are no FDA-approved therapies to treat FCS. Therefore, there is a critical unmet need for new FCS treatments that effectively lower triglyceride levels and prevent the complications associated with this disease.

If approved, olezarsen will be the first available treatment for FCS, a rare genetic disease that can lead to pancreatitis attacks. 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 who are eligible for treatment.