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The US Food and Drug Administration’s (FDA) Endocrinologic and Metabolic Drugs Advisory Committee recently voted in favor of approving Genzyme, a Sanofi (SNY - Analyst Report) company, and partner Isis Pharmaceuticals Inc.’s (ISIS - Analyst Report) candidate Kynamro (mipomersen). The panel voted 9 to 6 that sufficient data is available on Kynamro’s efficacy and safety profile for gaining final approval.  

The companies are seeking FDA approval for the use of Kynamro for the treatment of patients with homozygous familial hypercholesterolemia (HoFH). A response from the FDA is expected by January 29, 2013. Approval would trigger a $25 million milestone payment from Sanofi.

Kynamro is the lead pipeline candidate at Isis Pharma. Isis Pharma and Genzyme are also seeking approval for the candidate in the EU for HoFH and severe heterozygous familial hypercholesterolemia (HeFH).

Isis Pharma’s agreement with Genzyme dates back to 2008. As per the terms of the deal, Isis will not only receive commercialization and development milestones, it will also split net profits with Sanofi on Kynamro (70% – Sanofi, 30% - Isis) until worldwide sales eclipse $2 billion. Thereafter, the companies will split profits on Kynamro 50/50.

We note that the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee also reviewed Aegerion Pharmaceuticals, Inc.'s (AEGR - Analyst Report) lomitapide on October 17, 2012. The panel voted 13 to 2 in favor of approving lomitapide for reducing low-density-lipoprotein cholesterol (LDL-C), total cholesterol, apolipoprotein B, and triglycerides in adults with HoFH as an adjunct to a low-fat diet and other lipid-lowering therapies, with or without apheresis.

Although we believe Kynamro has blockbuster potential, we remain worried that concerns regarding the safety profile of the drug could limit its commercial potential once launched. Additional competition in form of lomitapide could also hamper the sales of Kynamro.

We currently have a Neutral recommendation on Isis Pharma.

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