Akcea Therapeutics and Ionis Pharmaceuticals announced that they received a Complete Response Letter (CRL) from the Food and Drug Administration (FDA) rejecting the New Drug Application for Waylivra (volanesorsen), an investigational treatment for familial chylomicronemia syndrome (FCS).

The CRL is from the FDA’s Division of Metabolism and Endocrinology Products Committee, who voted 12 to 8 in favor of approving Waylivra, in May of this year. The Company did not state the exact reasons given for the rejection, but CEO of Akcea, Paula Soteropoulos said, “We are extremely disappointed with the FDA’s decision. We continue to feel strongly that Waylivra demonstrates a favorable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward.” 

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Waylivra is designed to lower the production the ApoC-III, a protein produced in the liver that is involved in triglyceride regulation and other metabolic parameters. FCS is characterized by severe hypertriglyceridemia and the risk of unpredictable and potentially fatal acute pancreatitis. 

In May, the FDA’s Committee reviewed findings from two Phase 3 trials (APPROACH, COMPASS) and 1 open-label study (APPROACH) for Waylivra. Results from the APPROACH trial showed that compared to placebo patients who received Waylivra had a mean reduction in triglycerides of 77% from baseline. The most common adverse events were injection site reactions and reductions in platelet levels.

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