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Orphan Drugs And Rare Diseases: Global News (03.29.13)

March 29, 2013

Recent orphan drug and rare disease global developments are in the news. Here is a list and description of recent developments :

I – Japan

Genmab (Copenhagen, Denmark) and GlaxoSmithKline (GSK) announces March 25, 2013, the approval of Arzerra (Ofatumumab) by Japan’s Ministry of Health, Labour, and Welfare (MHLW) for the treatment of relapsed/refractory CD20-positive Chronic Lymphocytic Leukemia (CLL). The approval results in a milestone payment of DKK 20 million from GSK to Genmab. In 2009, the FDA approves orphan drug Arzerra for CLL. In Europe, the drug receives conditional approval for CLL.

II – Spain

A Spanish research consortium identifies the first mutation in a protein responsible for protecting telomeres (bits of DNA at the end of a chromosome), uncovering one of the most frequently mutated genes in Chronic Lymphocytic Leukemia (CLL). This finding “ … reveals that one of the three DNA-binding proteins at the telomere, known as protection of telomere 1 and coded for by the gene POT1, is a common mutation in CLL.” The discovery is published in the journal Nature Genetics.

III – Europe

The European Commission (EC) designates Mast Therapeutics’ MST-188 an orphan medicinal product for sickle-cell disease. This decision follows a positive opinion adopted by the Committee or Orphan Medicinal Products (COMP). Mast Therapeutics is a biopharmaceutical company in San Diego, California. The company also announces that it has engaged ESC Advisors to identify potential partners for MST-188.

A 4-year project (January 2013 – December 2016), RARE-Bestpractices, to identify best practices for the management of rare diseases is announced March 27, 2013. The project includes 14 partners across Europe and aims to improve the clinical management of rare diseases.

The European Medicines Agency (EMA) updates the Marketing Authorization and market exclusivity for orphan drugs. Please reference here for further detailed information.

IV – Canada

The Canadian government announces March 26, 2013, new personalized health research projects across Canada. This will offer “new hope for Canadian patients with debilitating diseases.” This is a result of a major $150-million genomics and personalized health competition, a Genome Canada-Canadian Institutes of Health Research (CIHR) partnership. 17 projects in total are approved for funding, each with a value in the range of $3 – $13 million. The projects focus on applying genomics to personalize patient treatments.

The Canadian Drug Expert Committee (CDEC) recommends that Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) be listed on the formulary listing of publicly funded drug plans for Cystic Fibrosis patients, age 6 and older, who have the G551D mutation.

V – United States

Edison Pharmaceuticals (Mountain View, CA) announces on March 28, 2013, that the company has signed a $545+ million licensing agreement with Japan’s Dainippon Sumitomo Pharma, for the development and commercialization of pediatric orphan inherited mitochondrial and adult central nervous system disease drugs.

Alexion Pharmaceuticals discloses, according to an article from, that the FDA issues a so-called 483 inspection report, which “refers to a failure to adhere to various good manufacturing practices”, for their Rhode Island facility that manufactures orphan drug Soliris. Also, in the same article, Alexion states that “.. the supply of Soliris to patients will not be interrupted and that the Warning Letter does not restrict production of Soliris or shipment of Soliris ….”.

Please Note: “Globe” by Augiasstallputzer at Wikimedia Commons [Public domain] | Wikimedia Commons.

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

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