Familial Chylomicronemia Syndrome Treatment to Get FDA Review
Akcea Therapeutics has submitted a New Drug Application (NDA) for volanesorsen, an investigational medicine for the treatment of familial chylomicronemia syndrome (FCS).
Volanesorsen is designed to reduce the production of ApoC-III, a protein produced by the liver that plays a central role in the regulation of plasma triglycerides. FCS is a rare disease that is characterized by extremely high levels of triglycerides which causes abdominal pain and can lead to acute pancreatitis.
The NDA submission is based on results from two Phase 3 studies (APPROACH and COMPASS). A total of 66 patients with FCS were enrolled in the year-long APPROACH study which met its primary endpoint of reduction in triglycerides at 3 months, with a 77% mean reduction in triglycerides for volanesorsen-treated patients.
The COMPASS study enrolled 113 patients with very high triglycerides (>500mg/dL) in a 6-month trial; it also achieved its primary endpoint, with a 71% mean reduction in triglycerides at 3-months in the volanesorsen-treated group compared to those in the placebo group.
“Today, there is no therapy for FCS patients that can effectively reduce triglycerides to levels that are even close to acceptable,” said Dr. Linda C. Hemphill of Massachusetts General Hospital and Harvard Medical School.
Volanesorsen had previously been granted Orphan Drug Designation for the treatment of patients with FCS. Paula Soteropoulos, CEO of Akcea said, “We are on track to launch volanesorsen globally in 2018 pending approval in the respective markets.”
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