Plozasiran for treating familial chylomicronaemia syndrome

Plozasiran is in clinical development for the treatment of patients with familial chylomicronaemia syndrome (FCS). FCS is a rare genetic disorder that causes very high levels of triglycerides, which are a kind of fat, in the blood. This can lead to serious health problems, including acute and chronic pancreatitis and diabetes. Symptoms of FCS include moderate to severe abdominal pain, unpredictable and recurrent episodes of acute pancreatitis, liver and spleen enlargement, reduced cognition, and fatigue. Current treatment options are limited, and an extremely restrictive, very low-fat diet is critical to help manage symptoms. This diet can be very challenging for patients, and often, many still have high triglyceride levels even when the diet is closely followed. Despite best intentions, patients, clinicians, and caregivers struggle with dietary control, and the risk of adverse consequences of raised triglycerides remains.