Isis Pharmaceuticals, Akcea Therapeutics Announce Start of Phase 3 Study of Volanesorsen in Patients with FPL

Isis Pharmaceuticals, Inc. ISIS and its wholly owned subsidiary, Akcea Therapeutics, today announced the start of a Phase 3 study to evaluate the efficacy and safety of volanesorsen in patients with familial partial lipodystrophy (FPL).  Akcea is currently conducting an international, multi-center Phase 3 study of volanesorsen in patients with Familial Chylomicronemia Syndrome (FCS).  Akcea is developing and plans to commercialize volanesorsen for two ultra-rare diseases, FCS and FPL. Familial partial lipodystrophy is a rare, genetic disorder characterized by metabolic abnormalities, including hypertriglyceridemia and extreme insulin resistance, and abnormalities in the distribution of body fat.  Patients with FPL have a high risk at an early age of diabetes, liver disease, and cardiovascular disease. In addition, extreme hypertriglyceridemia puts them at risk for potentially life-threatening pancreatitis.   Since it is most often an inherited, autosomal dominant disorder, FPL can be passed down from one generation to the next in affected families. "Every day, people with FPL struggle to manage the burden this disease brings across virtually every major organ system in their body," describes Dr. Elif Oral of the University See full press release