Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder caused by a mutation in lipoprotein lipase. FCS is characterized by the body’s inability to break down fats in the blood, which leads to the accumulation of chylomicrons. This can cause severe health complications such as pancreatitis and cardiovascular disease. One potential approach is the development of drugs that can specifically target the metabolic pathways involved in the accumulation of chylomicrons in FCS. With its ARO-ANG3, Arrowhead Pharmaceuticals is targeting unmet needs in rare lipid disorders, says GlobalData, a leading data and analytics company.

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).

Angiopoietin-like protein 3 (ANGPTL3) is a protein that regulates the activity of enzymes involved in the production and clearance of lipoproteins. There are currently several novel small molecule ANGPTL3 inhibitors in development, including Arrowhead Pharmaceuticals ARO-ANG3, which is being investigated for the treatment of homozygous FH with plans for Phase III clinical trials. This drug is expected to launch in the US in 2026 and reach $442 million in sales by 2028, forecasts GlobalData.

Dr Shireen Mohammad, Cardiovascular & Metabolic Disorders Analyst at GlobalData, comments: “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.”

Arrowhead Pharmaceuticals is also investigating ARO-APOC3 which is in a Phase II clinical study for patients with severe hypertriglyceridemia and mixed dyslipidemia, as well as a Phase III clinical study for patient with FCS. In March 2023, FDA granted Fast Track designation to ARO-APOC3 for reducing triglycerides in adult patients with FCS.  ARO-APOC3 is expected to launch in the US in 2026 and reach $178 million in sales by 2028, according to GlobalData.

Dr Mohammad concludes: “Overall, the development of drugs to treat severe genetic disorders has a significant, positive impact on individuals, families, and society as a whole, leading to better healthcare outcomes, improved quality of life, and advances in medical research.”