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Importance Of October 17, 2012 For Aegerion Pharmaceuticals’ Orphan Drug Lomitapide For Homozygous Familial Hypercholesterolemia (HoFH)

October 11, 2012

       What is the importance of October 17, 2012 for Aegerion Pharmaceuticals’ orphan drug Lomitapide ?    

       On October 17, 2012, the Endocrinologic & Metabolic Drugs Advisory Committee Meeting is scheduled to “discuss new drug application (NDA) 203858, lomitapide capsules, by Aegerion Pharmaceuticals, Inc. The proposed indication (use) is as an adjunct to a low-fat diet & other lipid-lowering drugs with or without LDL apheresis to reduce LDL cholesterol, total cholesterol, apolipoprotein B, and triglycerides in patients with Homozygous Familial Hypercholesterolemia (HoFH). (Apheresis is a laboratory technology used to remove LDL from the bloodstream)”. 

       Facts about Lomitapide :

  1. Receives FDA Orphan Drug Designation (ODD) October 23, 2007 for treatment of HoFH
  2. Receives FDA Orphan Drug Designation (ODD) March 3, 2011 for treatment of Familial Chylomicronemia

       Facts about Homozygous Familial Hypercholesterolemia (HoFH) : 

  1. Estimated 3,000 American patients
  2. Patients have inherited faulty genes from both parents that causes cholesterol management to malfunction; lack working receptors for LDL cholesterol which results in not being able to effectively clear LDL cholesterol from the blood

       William Tanner, an analyst with Lizard Capital Markets LLC in New York estimates that “lomitapide may draw $300 million or more in annual sales for Aegerion, based on a price of about $300,000/patient/year, similar to the cost of other drugs for diseases with few treatment options”.

References October 10, 2012 article titled,”Toddlers Facing heart Attacks Await Ruling on New Drugs”  

FDA 2012 Advisory Committee Tentative Meetings as of 10/10/2012

FDA 2012 Advisory Committee Calendar as of 10/10/2012 

Copyright © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.


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