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Orphan Drugs: Regulatory News From Europe (03.22.13)

March 22, 2013

Regulatory orphan drug developments from Europe are in the news today (Friday, 03/22/13). Here is a list and description of recent developments :

I – ARIAD Pharmaceuticals’ Iclusig (Ponatinib)

On March 22, 2013, ARIAD Pharmaceuticals announces that EMA’s Committee for Human Medicinal Products (CHMP) adopts a positive opinion on the Marketing Authorization Application (MAA) for Iclusig for two indications :

  1. Chronic Myeloid Leukemia (CML)
  2. Philadelphia chromosome-positive acute lymphoblastic Leukemia (Ph+ALL).

Harvey J. Berger, M.D., Chairman and CEO of ARIAD Pharmaceuticals says that, “ … we look forward to the anticipated marketing authorization by the European Commission around mid-2013.”

This EMA CHMP nod comes three months after the FDA, in December 2012, gives Accelerated Approval (following Priority Review)  for Iclusig for the treatment of CML and Ph+ALL. The FDA approval comes with a black box warning about the side effects of blood clots and liver toxicity. The approval comes more than 3 months before the FDA PDUFA date of March 27, 2013.

II– Genzyme And Isis Pharmaceuticals’ Kynamro (Mipomersen)

Isis Pharmaceuticals and Genzyme announces on March 22, 2013, that EMA’s CHMP confirms its previous position (December 2012) and is maintaining a negative opinion regarding the MAA for Kynamro for the treatment of Homozygous Familial Hypercholesterolaemia (HoFH). The FDA approves Kynamro in the US in January 2013 for HoFH.

Please reference a March 2013 Blog Post that documents the competition and race to market for Aegerion Pharmaceuticals’ Juxtapid (Lomitapide) and Kynamro for the same HoFH indication. This EMA CHMP rejection of Kynamro may negatively affect its position in the HoFH market space. Juxtapid’s price of $235,000-$295,000/year is much higher than Kynamro’s $176,000/year. It will be interesting to see how the competition for the HoFH market plays out.  

Please Note: “RSS” by Abzt at de.wikipedia (Transferred from de.wikipedia) [GPL] | Wikimedia Commons.

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

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