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Orphan Drugs And Rare Diseases: Global News (03.14.13)

March 14, 2013

Recent orphan drug and rare disease global developments are in the news. Please note that the next global news Blog Post will include Asia and Latin America. Here is a list and description of recent developments :

I – Ukraine

A March 12, 2013 article in Interfax News Wire, says that the Ukrainian government, according to a senior health care official, will provide free drugs to rare disease patients starting in 2014. Tetiana Bakhteyeva, head of a committee on health, says that at least 30% of rare disease patients die in childhood before the age of 5. The Ukraine, which does not have any official data or statistics on rare diseases will create a list of rare diseases and will also register patients with these illnesses. Ms. Bakhteyeva says that there is only data on certain groups of sick children who are given drugs using state budget funds :

  1.   16 with Gaucher Disease
  2. 573 with Cystic Fibrosis
  3. 618 with Pituitary Dwarfism
  4. 370 with Hemophilia
  5.   91 with Congenital Immunodeficiency
  6. 195, < 3 years of age, with Phenylketonuria.

The Ukrainian authorities are currently providing drugs to about 1,860 rare disease patients.

II – Australia

In Australia, the administrative authority involved in orphan drug regulations, is the Therapeutic Goods Administration (TGA). TGA is part of the Australian government’s Department of Health & Ageing. The TGA designates on March 4, 2013, Bayer’s Nexavar (Sorafenib) as an orphan drug for the treatment of patients with radioactive iodine treatment for refractory, locally advanced or metastatic Differentiated Thyroid Cancer (DTC).

Pharmaxis, a biopharmaceutical company, promotes COO Gary Phillips to the post of CEO, effective immediately. Mr. Phillips replaces founding CEO Dr. Alan Robertson. In January 2013, the FDA Advisory Committee did not recommend orphan drug Bronchitol for approval for Cystic Fibrosis. Bronchitol has Orphan Drug Designation in the US, Europe, and Australia. It is approved for marketing in Australia and throughout the European Union.

The alphabetical list of designated orphan drugs in Australia from the TGA is updated as of January 30, 2013.

III – Europe

The European Medicines Agency (EMA) grants orphan drug status to Activartis’ cancer immunotherapy AV0113 for the treatment of glioma.

Juan Carrion, President of the Spanish Federation for Rare Diseases (FEDER), proposes to the Spanish government, a set of 13 measures for 2013, with the purpose of improving the quality of life and providing support for rare disease patients. The 13 measures are a result of over 250 associations of patients with rare diseases. The list of initiatives includes promoting research on rare diseases, “declaring them chronic diseases, and exempting affected families from pharmaceutical copayment”.

Sweden’s Premacure is acquired by Shire. Premacure is developing a treatment for a potentially blinding eye disorder that affects premature babies. This is another opportunity for Shire to continue to expand their rare disease franchise.

On March 12, 2013, it is announced that the National Health Service (NHS) in Northern Ireland is to provide Vertex Pharmaceuticals’ orphan drug Kalydeco (Ivacaftor) for patients with Cystic Fibrosis (CF), ages 6+ and who have at least one copy of the G551D mutation.

IV – Canada

Alexion Pharma Canada’s Soliris (Eculizumab) receives Health Canada’s approval for the treatment of Atypical Hemolytic Uremic Syndrome (aHUS). Soliris is the 1st and only therapy for aHUS.

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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