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What Is The Status Of Genzyme And Isis Pharmaceuticals’ Orphan Drug Kynamro (Mipomersen) In The EU ?

December 15, 2012

It is important to note that Kynamro (Mipomersen) has FDA Orphan Drug Designation (ODD) but no orphan designation in the EU. Kynamro is for the treatment of Homozygous Familial Hypercholesterolemia (HoFH).

Genzyme and Isis Pharmaceuticals announces that Kynamro is issued on December 13, 2012, a  negative opinion by the European Medicines Agency’s (EMA) Medicinal Products for Human Use (CHMP), for its Marketing Authorization Application (MAA). Genzyme can ask for a re-examination of the opinion within 15 days of notification of this negative opinion. Genzyme plans on requesting a re-examination of the CHMP Opinion.

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 …. ”.

Here is a FDA historical timeline of Kynamro (Mipomersen) :

  1. On October 18, 2012, the FDA ‘s Endocrinologic & Metabolic Drug Advisory Committee recommends, in a 9-6 vote, Genzyme and Isis Pharmaceuticals’ orphan drug Kynamro (Mipomersen) for the treatment of Homozygous Familial Hypercholesterolemia (HoFH). The recommendation is reached despite concerns about side-effects, such as liver problems and a possible higher risk for cancer
  2. Upcoming FDA PDUFA date :
Generic Name Trade Name Sponsor Indication PDUFA Date
Mipomersen Sodium Kynamro Genzyme/Isis Pharmaceuticals Homozygous Familial Hypercholesterolemia (HoFH) 1/29/13

It is important to keep in mind the followig :

  1. Aegerion Pharmaceuticals’ orphan drug Lomitapide for the treatment of HoFH, receives a slightly stronger approval recommendation by the same FDA Advisory Committee in October 2012.
  2. Lomitapide receives orphan designation on December 17, 2010, from EMA’s COMP.


Bloomberg’s December 14, 2012 article titled, “Sanofi cholesterol drug with Isis fails to win EU backing”

EMA December 13, 2012 Q&A Document on the refusal of the marketing authorization for Kynamro December 14, 2012 article titled, “CHMP recommends against approval for Mipomersen in Europe” December 14, 2012 article titled, “Read the European Medicines Agency’s Smackdown of Kynamro”.

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

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