The US Food and Drug Administration (FDA) recently approved Aegerion Pharmaceuticals’ (AEGR - Analyst Report) Juxtapid (lomitapide) capsules. Juxtapid has been approved for the treatment of patients suffering from homozygous familial hypercholesterolemia (HoFH) as an adjunct to a low-fat diet and other lipid-lowering therapies.
The FDA’s decision was based on positive results from a phase III study on Juxtapid. Aegerion evaluated the safety and effectiveness of the drug in reducing low-density lipoprotein cholesterol (LDL-C) levels in 29 adults suffering from HoFH, in the study.
Results from the study revealed that Juxtapid when added to existing lipid-lowering therapy resulted in a 40% reduction in LDL-C in HoFH patients who discontinued the study prematurely at week 26. The same was reduced by 50% in 23 patients who completed the study through week 26.
We note that Juxtapid’s label carries a boxed warning regarding the risk of liver toxicity. The drug is available through the Juxtapid Risk Evaluation and Mitigation Strategy (REMS) Program due to the risk of liver toxicity.
Aegerion will be conducting an observational cohort study on the drug for the long-term safety and effectiveness of the drug, as per its commitment to the FDA.
We are encouraged with the approval of Juxtapid. We note that companies like Isis Pharmaceuticals and Sanofi (SNY - Analyst Report) are also looking to enter the market with their HoFH candidate, Kynamro, which is currently under review in the US.
We currently have a Neutral recommendation on Aegerion. The stock carries a Zacks #3 Rank (Hold). Other stocks in the pharma sector that carry a Zacks #1 Rank (Strong Buy) are Targacept and Repligen (RGEN - Snapshot Report) .