europeanpharmaceuticalreviewMarch 24, 2017
Tag: familial chylomicronaemia syndrome
Akcea Therapeutics',a wholly owned subsidiary of Ionis Pharmaceuticals pivotal Phase 3 study of volanesorsen met its primary endpoint of reducing triglyceride levels in patients with familial chylomicronaemia syndrome (FCS).
The study is a randomised, double-blind, placebo-controlled, 52-week Phase 3 study in 66 patients with FCS, a rare disease affecting approximately 1-2 in every one million people in the European Union. The mean baseline triglyceride level of patients in the study was 2,209 mg/dL. Patients treated with volanesorsen experienced robust reductions in triglycerides and related benefits as follows:
"We are excited about the strong profile of volanesorsen in not only robustly reducing triglycerides, but also providing additional important patient benefits. FCS is a life-threatening, rare disease with multiple severe daily and chronic manifestations. We believe the efficacy and safety data from volanesorsen studies demonstrate a favourable risk-benefit profile for patients with FCS," said Paula Soteropoulos, president and chief executive officer, Akcea Therapeutics.
The study will support the regulatory submission for familial chylomicronaemia syndrome of volanesorsen. Additional data from the study will be presented at an upcoming medical meeting.
"The success of the study represents an important milestone towards our first regulatory submissions for volanesorsen in the US, Europe and Canada in 2017," said Dr Louis O’Dea, chief medical officer, Akcea Therapeutics. "We seek to bring this new treatment as expeditiously as possible to FCS patients who have a high unmet need with potentially life-threatening consequences."
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