Volanesorsen Designated Orphan Drug Status for Familial Chylomicronemia Syndrome

Akcea Therapeutics announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS).

Patients with FCS treated with volanesorsen achieved substantial reductions in apoC-III, triglycerides, chylomicrons and apoC-III-associated very low density lipoprotein-cholesterol (VLDL-C) particles as observed in a Phase 2 study. Akcea is conducting an international multi-center, randomized, double-blind, placebo-controlled Phase 3 study in patients with FCS.

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Volanesorsen is an antisense drug designed to target apoC-III. It is currently in development to treat patients with severely high triglycerides either as a single agent or in combination with other triglyceride-lowering agents.

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