Aegerion Conducts Phase III Trial For Anti-Cholesterol Drug

The clinical trial will test the efficacy of lomitapide as an adjunct treatment to reduce low-density lipoprotein cholesterol (LDL-C) in patients suffering from homozygous familial hypercholesterolemia.

AsianScientist (Apr. 16, 2014) – Aegerion Pharmaceuticals, Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for patients with debilitating rare diseases, has embarked on a Phase III clinical trial for homozygous familial hypercholesterolemia (HoFH) in Japan.

The open-label trial will test to efficacy of the drug lomitapide as an adjunct treatment to reduce low-density lipoprotein cholesterol (LDL-C). Lomitapide received orphan drug designation from Japan’s Ministry of Health, Labor and Welfare last year.

Estimated enrollment is between five and ten adult HoFH patients who are receiving concomitant lipid-lowering therapies. 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 Japanese regulatory authorities have indicated they will allow the new drug application to be filed following completion of the 26 week efficacy phase.

“This pivotal trial marks an important milestone for Aegerion and Japanese HoFH patients,” said Dr. Mark Sumeray, Aegerion’s Chief Medical Officer. “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.”

“Initiation of the Phase III trial in Japan allows us to sharpen focus on our regulatory timeline as we seek approval in this key market,” said Martha Carter, Chief Regulatory Officer of Aegerion. “We currently hope to submit the filing for marketing approval in Japan as early as mid-2015.”

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.

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Source: Aegerion Pharmaceuticals Inc.

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