US-based Aegerion Pharmaceuticals has started patient enrolment in Japan for a Phase III clinical trial of lomitapide as an adjunct treatment to reduce low-density lipoprotein cholesterol (LDL-C) in Japanese patients with homozygous familial hypercholesterolemia (HoFH).
The company said that the open-label Phase III trial in Japan is similar in design to the completed Phase III trial carried out in the US and other countries.
About five and ten adult HoFH patients who are receiving concomitant lipid-lowering therapies including apheresis are expected to be enrolled in the Phase III trial in Japan.
Following a six week run-in period, HoFH patients will be given lomitapide for 26 weeks, starting at 5mg/day and escalating to a maximum dose of 60mg based on tolerability, and after 26 weeks, patients will enter a 30 week safety phase.
The trial’s primary endpoint is percentage change in LDL-C levels from baseline to week 26, while secondary endpoints include percentage change in lipid parameters, long-term safety, and changes in hepatic fat from baseline to week 56.
According to the Japanese regulatory authorities (PMDA), the company will be allowed to file a new drug application (NDA) after completion of the 26 week efficacy phase.
Aegerion chief medical officer Mark Sumeray said the pivotal trial marks an important milestone for the company and Japanese HoFH patients.
"Japan is a well-characterised region for patients with severe hypercholesterolemia and investigators there have shown a high level of enthusiasm for the trial, recognising the unmet clinical need for their patients living with this serious disease," Sumeray said.
The company expects to submit the filing for marketing approval in Japan as early as mid-2015.
In 2013, the company received orphan drug designation from Japan’s Ministry of Health, Labour and Welfare for lomitapide.
HoFH is inherited from both parents that impairs the function of the receptor responsible for removing LDL-C (bad cholesterol) from the body.
Image: Microphotography of arterial wall with calcified (violet colour) atherosclerotic plaque. Photo: courtesy of Kwz.