Updated on 11 April 2014
The phase III, open-label trial in Japan is similar in design to the completed Phase III trial conducted in the United States and other countries
Singapore: Leading biopharmaceutical company Aegerion Pharmaceuticals that is dedicated to the development and commercialization of novel therapies for patients with debilitating rare diseases has said that it is going to begin phase III clinical trials in Japan.
The company said that patient enrollment has been initiated in Japan in a clinical trial of lomitapide as an adjunct treatment to reduce low-density lipoprotein cholesterol (LDL-C) in Japanese patients with homozygous familial hypercholesterolemia (HoFH).
Last year, Aegerion received orphan drug designation from Japan's Ministry of Health, Labour and Welfare for lomitapide.
The phase III, open-label trial in Japan is similar in design to the completed Phase III trial conducted in the United States and other countries. Estimated enrollment is between five and ten adult HoFH patients who are receiving concomitant lipid-lowering therapies, including, in some cases, apheresis.
After a six week run-in period, patients will receive lomitapide for 26 weeks, starting at 5 mg/day and escalating to a maximum dose of 60 mg based on tolerability. After 26 weeks, patients will enter a 30 week safety phase. The primary endpoint is % change in LDL-C levels from baseline to week 26. Secondary endpoints include % change in lipid parameters, long-term safety, and changes in hepatic fat from baseline to week 56. The Japanese regulatory authorities (PMDA) have indicated they will allow the new drug application to be filed following completion of the 26 week efficacy phase.