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Why Is Next Week Important For Genzyme and Isis Pharmaceuticals’ Orphan Drug Kynamro ?

January 23, 2013

Next Tuesday, January 29, 2013, is the scheduled FDA PDUFA date for Genzyme and Isis Pharmaceuticals’ orphan drug Kynamro (Mipomersen) for the treatment of Homozygous Familial Hypercholesterolemia (HoFH) :

Generic Name Trade Name Sponsor Indication PDUFA Date
Mipomersen Sodium Kynamro Genzyme/Isis Pharmaceuticals Homozygous Familial   Hypercholesterolemia (HoFH) 1/29/13

Here is a regulatory timeline for Kynamro :

1)  May 23, 2006 : Receives FDA Orphan Drug Designation (ODD); no orphan designation in the EU

2)  October 18, 2012 : The FDA ‘s Endocrinologic & Metabolic Drug Advisory Committee recommends, in a 9-6 vote, Kynamro (Mipomersen) for HoFH. The recommendation is reached despite concerns about side-effects, such as liver problems and a possible higher risk for cancer

3)  December 13, 2012 : Genzyme and Isis Pharmaceuticals announces that Kynamro is issued a negative opinion by the European Medicines Agency’s (EMA) Medicinal Products for Human Use (CHMP), for its Marketing Authorization Application (MAA). The EMA CHMP’s decision is based on their concern about the drug’s safety, noting “ ….. that a high proportion of patients stopped taking the medicine within 2 years, mainly due to side effects ….. This was considered important because Kynamro is intended for long-term treatment in order to maintain the cholesterol-lowering effect. CHMP was also concerned by liver test results … showing a build-up of fat in the liver and increased enzyme level, and was not convinced that the company had proposed sufficient measures to prevent the risk of irreversible liver damage …. ”. Isis Pharmaceuticals per the presentation given on January 7, 2013, at the J.P. Morgan Healthcare Conference, anticipates a new EMA CHMP opinion in the 2Q 2013

4)  January 7, 2013 : Isis Pharmaceuticals gives a presentation at the 31st Annual J.P. Morgan Healthcare Conference

Kynamro and Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapide) are in a battle for the same indication in the United States, HoFH. Both orphan drugs have been nearly parallel in development in the pipeline. Both drugs are proven effective, which leaves the question of safety. Below is a brief outline of Kynamro’s competitor :

Juxtapid (Lomitapide)

1) Receives FDA approval on 12/21/12

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

3) Has FDA ODD & Orphan Designation in the EU

5) Once-daily pill vs Kynamro’s weekly injection.

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 HoFH 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”

PharmaLive.com article titled, “Genzyme Awaits FDA Decision on Kynamro”.

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

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