Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder caused by a mutation in lipoprotein lipase. FCS is characterised 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.

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 (FH). There are currently no FDA-approved therapies to treat FCS.

One unmet need in the treatment of FCS is the development of new therapies that can more effectively lower triglyceride levels and prevent the complications associated with this disorder. One potential approach is the development of drugs that can specifically target the metabolic pathways involved in the accumulation of chylomicrons in FCS.

Angiopoietin-like protein 3 (ANGPTL3) is a protein that regulates the activity of enzymes involved in the production and clearance of lipoproteins. There are a number of novel small molecule ANGPTL3 inhibitors in development, including Arrowhead Pharmaceuticals’ ARO-ANG3, currently being investigated for the treatment of homozygous FH with plans for Phase III clinical trials.

GlobalData forecasts this drug to launch in the US in 2026 and to reach $442 million in sales by 2028. Arrowhead Pharmaceuticals is also investigating ARO-APOC3, currently in a Phase II clinical study for patients with severe hypertriglyceridemia and mixed dyslipidemia, as well as in a Phase III clinical study for patients with FCS.

In March 2023, Arrowhead Pharmaceuticals announced that the FDA has granted Fast Track designation to ARO-APOC3 for reducing triglycerides in adult patients with FCS. Fast Track designation is a process designed to facilitate the development of drugs to treat serious conditions and fulfil unmet needs, and to allow new drugs to reach patients earlier. GlobalData forecasts that ARO-APOC3 will launch in the US in 2026 and reach $178 million in sales by 2028.

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.