Updated on 11 April 2014
"This pivotal trial marks an important milestone for Aegerion and Japanese HoFH patients. Japan is a well-characterized region for patients with severe hypercholesterolemia and investigators there have shown a high level of enthusiasm for the trial, recognizing the unmet clinical need for their patients living with this serious disease," said Mr Mark Sumeray, MD, Chief Medical Officer.
"Initiation of the phase three trial in Japan allows us to sharpen focus on our regulatory timeline as we seek approval in this key market. We currently hope to submit the filing for marketing approval in Japan as early as mid-2015," said Ms Martha Carter, Chief Regulatory Officer.
HoFH is a serious, rare genetic disease inherited from both parents that impairs the function of the receptor responsible for removing LDL-C (‘bad' cholesterol) from the body. A loss or impairment of LDL receptor function results in severe elevation of blood cholesterol levels. HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.