Skip to content

Orphan Drugs And Rare Diseases: Global News (03.21.13)

March 21, 2013

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

I – Russian Federation

Per a March 19, 2013 article, the Russian Federation’s Ministry of Health publishes in late January 2013, draft amendments to the country’s drug registration process. Some of the proposed draft amendments have an impact on orphan drugs :

  1. Accelerated registration for orphan drugs and newly registered generics. The new acceleration procedure will not be applicable to biosimilars, originator drugs (except orphan drugs), and other types of drugs.
  2. For orphan drugs “the results of preclinical and clinical studies performed outside of the Russian Federation in accordance with the rules of good laboratory practice and good clinical practice will be recognized.”

The implications of both the accelerated registration process and the recognition of results of preclinical/clinical trials performed outside of Russia, are a positive development for orphan drug manufacturers. It is a positive development because of the issues and challenges that face conducting domestic clinical trials in Russia –a limited rare disease patient population. The review of the draft amendments is expected to continue until March 23.  

II – Brazil

Protalix BioTherapeutics and Pfizer announces March 18, 2013, that Brazil’s National Health Surveillance Agency, ANVISA (Agencia Nacional de Vigilancia Sanitaria), grants regulatory approval to Uplyso (known as Elelyso outside of Latin America) for Type I Gaucher Disease in adults. This approval follows Uruguay’s Ministry of Public Health’s approval of the drug in November 2012.

III – Australia

The FDA on March 18, 2013, concludes its review of the New Drug Application (NDA) for Pharmaxis’ orphan drug Bronchitol (Mannitol) for Cystic Fibrosis (CF).  The FDA recommends that Pharmaxis should conduct an additional clinical trial to obtain approval. Bronchitol has Orphan Drug Designation in the US, Europe, and Australia. It is approved for marketing in Australia and throughout the EU. Bronchitol was discovered and developed in Australia. 

The Federal Court of Australia dismisses a 2010 lawsuit to prevent the patenting of the BRCA1 and BRCA2 genes. This ruling in favor of Myriad Genetics and Genetic Technologies, upholds the decision to allow patenting of human genes. An appeal for this decision is filed in the Federal Court of Appeals in Australia.   

IV – Asia-Pacific

In 2012, the Asia Pacific Orphan Drug Alliance (APODA) is formed, to provide one organization for physician and patient access to orphan drugs in 9 countries with a total population of approximately 500 million people. It is an alliance of independent companies formed to “overcome the barriers to orphan drug access across the Asia-Pacific region. These barriers traditionally include a complex and diverse regulatory environment and uncertainty over government or patient ability to pay …  in multiple territories.” APODA offers orphan drug expertise covering this part of the globe. Please reference the website for further information about this organization.

According to a February 2013 China news article, seventeen medical institutions launch China’s first national organization against rare diseases, the China Rare Diseases Prevention and Treatment Alliance. The new organization’s objectives are to :

  1. Establish rare disease treatment centers
  2. Research on the cure for rare diseases
  3. Provide publicity and medical education
  4. Assist with rare disease data collection and epidemiological studies.

The World Orphan Congress Asia is to be held in Singapore, June 18 – 19, 2013.

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

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

Leave a Comment

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out / Change )

Twitter picture

You are commenting using your Twitter account. Log Out / Change )

Facebook photo

You are commenting using your Facebook account. Log Out / Change )

Google+ photo

You are commenting using your Google+ account. Log Out / Change )

Connecting to %s

%d bloggers like this: