The European Commission has approved Aegerion Pharmaceuticals' Lojuxta (lomitapide) hard capsules as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with homozygous familial hypercholesterolemia (HoFH).

"The approval of Lojuxta marks an important day for patients in Europe with HoFH, and is a major step for Aegerion in our goal to bring therapy to patients globally," said Marc D Beer, chief executive officer of Aegerion. "We are now keenly focused on launch, and are actively working with national health authorities in an effort to make Lojuxta available as swiftly as possible."

"HoFH is a rare and serious medical condition," said Prof. Alberico L Catapano, president of the European Atherosclerosis Society. "Despite the treatments we have available today, patients continue to experience severe elevations in LDL-C. With novel therapies such as Lojuxta it is possible to achieve target levels in LDL-C for patients with HoFH. This marks a change in our ability in daily practice to truly impact the lives of these patients."

The Summary of Product Characteristics (SPC) describes that genetic confirmation of HoFH should be obtained whenever possible, and that other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolemia (e.g., nephrotic syndrome, hypothyroidism) must be excluded.

The European Commission's decision is based on a favourable benefit-risk assessment from Aegerion's phase III study which evaluated the safety and efficacy of the medicine to reduce LDL-C levels in 29 adult patients with HoFH.

When added to the existing lipid-lowering therapy of the HoFH patients in the study, Lojuxta significantly reduced LDL-C from a baseline average of 336 mg/dL to 190 mg/dL (40 per cent reduction) at Week 26 in the intent-to-treat population. LDL-C was reduced by an average of 50 per cent in the 23 patients who completed the study through Week 26. After Week 26, during the safety phase of the study, adjustments to concomitant lipid-lowering treatments, including apheresis, were allowed. Average reductions in LDL-C were sustained during chronic therapy and no additional dropouts were seen.

The most common adverse reactions in the phase III trial were gastrointestinal, reported by 27 (93 per cent) of 29 patients. Diarrhoea occurred in 79 per cent of patients, nausea in 65 per cent, dyspepsia in 38 per cent, and vomiting in 34 per cent. Other reactions reported by at least 20 per cent of patients include abdominal pain, abdominal discomfort, abdominal distension, constipation, and flatulence. Elevations in liver enzymes and hepatic (liver) fat were also observed.

Ten of the 29 patients in the study had at least one elevation in liver enzymes greater than or equal to three times the upper limit of normal, including four patients who experienced liver enzymes greater than or equal to five times the upper limit of normal. Liver enzyme elevations were managed through dose reduction or temporary discontinuation of dose. There were no clinically meaningful elevations of total bilirubin, international normalized ratio (INR) or alkaline phosphatase, which are other markers of potential harmful effects on the liver. Hepatic fat increased from a baseline of one percent to a median absolute increase of six percent at 26 and 78 weeks.

Aegerion has adopted a risk management plan to help educate physicians on the safety information for Lojuxta and appropriate precautions to be followed by healthcare professionals and patients. In addition, as part of Aegerion's post-marketing commitment to both the FDA and the EMA, the Company will conduct an observational cohort study to generate more data on the long-term safety profile of lomitapide, the patterns of use and compliance and the long-term effectiveness of controlling LDL-C levels.

Aegerion Pharmaceuticals is dedicated to the development and commercialization of innovative, life-altering therapies for patients with debilitating, often fatal, rare diseases.