Familial Chylomicronemia Syndrome

The FDA has granted fast track designation for olezarsen in the treatment of familial chylomicronemia syndrome, according to a press release from Ionis Pharmaceuticals.

While rare, certain genetic and multifactorial causes of severe hypertriglyceridemia may warrant special considerations for diagnosis and treatment within their respective patient populations, a speaker reported.

In a phase 2 study, evinacumab reduced triglyceride levels in patients with severe hypertriglyceridemia but not familial chylomicronemia syndrome, a speaker reported at the American College of Cardiology Scientific Session.

Akcea Therapeutics and Ionis Pharmaceuticals announced that a phase 2 study of AKCEA-APOCIII-LRx for the treatment of patients with hypertriglyceridemia at risk for or with established CVD met its primary endpoint of significant triglyceride lowering.

Friday marks the second annual Familial Chylomicronemia Syndrome Awareness Day, a day dedicated to raising global observance of the challenges associated with the rare genetic disease and the critical need for treatment.