Familial Chylomicronemia Syndrome

March 13, 2017

Akcea Therapeutics issued results of a phase III study of volanesorsen for reducing triglyceride levels in subjects with familial chylomicronemia syndrome (FCS). APPROACH was a randomized, double-blind, placebo-controlled, 52-week study in 66 patients with FCS, a rare disease affecting approximately 3,000 to 5,000 patients worldwide. The average incoming triglyceride level of patients in the study was 2,209mg/dL. For the primary endpoint of the study, volanesorsen-treated patients (n=33) achieved a statistically significant (p<0.0001) mean reduction in triglycerides of 77% from baseline after three months of treatment, compared to a mean increase of 18% in placebo-treated patients (n=33). This represented a mean absolute reduction of 1,712mg/dL in treated patients. Of the treated patients who entered the study with triglycerides 750mg/dL, 50% achieved triglyceride levels less than 500mg/dL after three months of treatment. By comparison, none of the placebo-treated patients achieved this level (p<0.003). Volanesorsen-treated patients with the highest documented frequency of pancreatitis attacks suffered no attacks during the 52-week treatment period (p=0.02). The APPROACH study will support the regulatory submission for FCS of volanesorsen.

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