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Aegerion Pharmaceuticals’ Lomitapide Verses Genzyme’s Mipomersen: Battle Of Two Orphan Drugs

December 18, 2012

Two orphan drugs, Aegerion Pharmaceuticals’ Lomitapide and Genzyme and Isis Pharmaceuticals’ Kynamro (Mipomersen), are in a battle for the same indication in the United States – Homozygous Familial Hypercholesterolemia (HoFH). Both orphan drugs have been nearly parallel in development in the pipeline.  Both drugs are proven effective, which leaves the question of safety.

Here are the comparisons between both drugs :

Lomitapide

1)     FDA PDUFA date on or about 12/28/12

2)     FDA’s Endocrinologic & Metabolic Drug Advisory Committee recommends unanimously for HoFH

3)     Has FDA Orphan Drug Designation (ODD)

4)     Has Orphan Designation in EU

5)     Once-daily pill.

Kynamro (Mipomersen)

1)     FDA PDUFA date of 01/29/13

2)     FDA’s Endocrinologic & Metabolic Drug Advisory Committee recommends, 9 to 6, for HoFH, but safety profile is of concern

3)     Has FDA ODD

4)     Doesn’t have Orphan Designation in EU

5)     Negative recommendation is issued by European Medicines Agency’s (EMA) Medicinal Products for Human Use (CHMP) for its Marketing Authorization Application (MAA) on December 13, 2012. Safety concerns such as cardiovascular risks and liver toxicity in trials were identified. Genzyme is working to have this decision re-examined & a final opinion should be made in the 2nd Quarter 2013.

LifeSci Advisors LLC, a provider of investment research for the life sciences sector, did a survey of 39 lipidologists who identified, if the FDA approves both medications what  their use of Lomitapide and Mipomersen in patients with HoHF would be. Here are the findings from this survey :

  1. 44% of the responding lipidologists view Lomitapide as more efficacious compared to 13% for Mipomersen
  2. 62% of the responding lipidologists view Lomitapide as “moderately safe” compared to 43% for Mipomersen.

References

LifeSciAdvisors.com July 2, 2012 article titled, “Survey Shows Strong Preference for Lomitapide over Mipomersen”

The Motley Fool December 14, 2012 article titled, “Aegerion Hits New High”

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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