As it goes about launching Waylivra (volanesorsen) in Europe, the Ionis Pharmaceuticals (Nasdaq: IONS) affiliate Akcea Therapeutics (Nasdaq: AKCA) has announced the publication of data on the rare disease therapy in The New England Journal of Medicine (NEJM).
Final results from the Phase III APPROACH study have been published in the journal, showing Waylivra’s promise in familial chylomicronemia syndrome (FCS), a debilitating disease caused by impaired production or function of the enzyme, lipoprotein lipase, responsible for breaking down chylomicrons, lipoproteins rich in triglycerides.
Results from the APPROACH study indicate that Waylivra lowers triglyceride levels below the threshold for risk of triglyceride-induced acute pancreatitis in the majority of patients.
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