Phase 3 Study Testing Volanesorsen in Familial Partial Lipodystrophy Begins

James Radke, PhD

A Phase 3 study to test the efficacy and safety of volanesorsen in patients with familial partial lipodystrophy (FPL) has started. Today, Isis Pharmaceuticals, and its subsidiary Akcea Therapeutics announced the trial has begin.

Familial partial lipodystrophy is a rare, genetic disorder in which patients are unable to store fat or triglycerides in normal fat stores so excess triglycerides builds up in various organs and the blood. 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.  

In a news release, Elif Oral, MD, of the Inuversity of Michigan said, "Every day, people with FPL struggle to manage the burden this disease brings across virtually every major organ system in their body."  Dr. Oral added, "FPL takes an enormous toll on those affected – both physically and psychologically. There is still no pharmacologic therapy developed specifically for this patient population.  For these patients in particular, the potential benefit of inhibiting the synthesis of apoC-III is very promising." 

Volanesorsen is an antisense drug designed to reduce the production of apoC-III, a protein that acts as a key regulator of triglyceride levels in the blood. The drug is also in a phase 3 study in patients with familial chylomicronemia syndrome.

The Phase 3 study announced today is a randomized, double-blind, placebo-controlled, multi-center, international study in approximately 50 patients with FPL given 300 mg once weekly dose of volanesorsen or placebo over 12 months. The primary endpoint is percent change in fasting triglycerides from baseline after 3 months of dosing.

For more information about the trial, visit
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