The US Food and Drug Administration's (FDA) Endocrinologic and
Metabolic Drugs Advisory Committee recently voted in favor of
approving Genzyme, a
) company, and partner
Isis Pharmaceuticals Inc.'s
) 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
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
) 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
We currently have a Neutral recommendation on Isis Pharma.
AEGERION PHARMA (AEGR): Free Stock Analysis
ISIS PHARMACEUT (ISIS): Free Stock Analysis
SANOFI-AVENTIS (SNY): Free Stock Analysis
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