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Orphan Drugs And Rare Diseases: European News (07.20.13)

July 20, 2013

Orphan Drug Designation (ODD) in the European Union (EU) is given to products that are designed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious. A disease is defined as rare in the EU if it affects fewer than five in 10,000 people. Granting of ODD in the EU, provides companies with development and commercial incentives:

•   A period of market exclusivity
•   Access to a centralized review process
•   Protocol assistance (scientific advice)
•   Waiving of marketing and post-marketing authorization fees.

Here are a few of the recent orphan drug and rare disease developments in Europe.

Alexion Pharmaceuticals announces on July 16, 2013, that the European Medicines Agency’s (EMA) COMP (Committee for Orphan Medicinal Products), gives a positive opinion for ODD for Soliris (Eculizumab) for the treatment of Neuromyelitis Otica (NMO). Soliris receives in June 2013, a FDA ODD, for the same indication.   NMO, also known as Devic’s Disease, involves an attack of the spinal cord and optic nerves by the body’s own immune system. Soliris is currently being evaluated in an investigator-initiated Phase 2 trial in patients with NMO.

Soliris is Alexion Pharmaceuticals’ only marketed product and generates blockbuster profits from multiple indications for ultra-rare diseases. Soliris is one of the world’s most expensive drugs, costing approximately $409,500/year/patient. Soliris treats ultra-rare diseases such as :

•   Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare genetic blood disorder (approved in 40 countries)
•   Atypical Hemolytic Uremic Syndrome (aHUS), an ultra-rare genetic disorder (approved in US and EU).

On July 17, 2013, the European Commission (EC) grants Topotarget, a Scandinavian-based clinical development oncology company, ODD for the treatment of malignant thymomas. Malignant thymomas are very rare tumors with a prevalence of 0.13 – 0.25 per 100,000. There is no product authorized currently in the EU for the treatment of malignant thymomas. In October 2012, the EU grants Belinostat ODD for Peripheral T-Cell Lymphoma.

OXiGENE, a clinical-stage biopharmaceutical San Francisco company developing novel therapeutics to treat cancer, announces on July 18, 2013, that the EMA grants ODD for Zybrestat (Fosbretabulin Tromethamine) for ovarian cancer. Data from a randomized, two-arm Phase 2 clinical trial testing the combination of Zybrestat and Avastin (Bevacizumab) to treat patients with advanced ovarian cancer could be available in early 2014, and, if positive, could provide the basis for a registration program.

Two of the three EU ODDs involve an oncology indication, which make up 40% of orphan drug indications.

NHS England announces on July 17, 2013, that they are seeking patient and public voice representatives (PPV) for the Rare Diseases Advisory Group (RDAG). Recommendations are for specialized services or technologies that should be considered for investment – such as Enzyme Replacement Therapy (ERT). RDAG is formed for the purpose of making recommendations to NHS in:

•   England
•   Scotland
•   Wales
•   Northern Ireland.

EURORDIS (Rare Diseases Europe) recently signs a Memorandum of Understanding with the Japan Patients Association (JPA), uniting patient advocates from Japan and Europe to promote rare diseases as an international health priority.

Per the July 18, 2013 online OrphaNews Europe website, the 2013 edition of the annual report on the State of the Art of Rare Disease Activities in Europe is now available online:

•   Part I – Overview of Rare Disease Activities in Europe
•   Part II – Key Developments in the Field of Rare Diseases in Europe in 2012
•   Part III – European Commission (EC) Activities in the Field of Rare Diseases
•   Part IV – European Medicines Agency (EMA) activities and other European Activities in the Field of Rare Diseases
•   Part V – Activities of European Member States and other European Countries in the Field of Rare Diseases.

Please Note: “Erlenmeyer Flasks” From Argonne US National Lab  [Public domain in the US] | Wikimedia Commons.

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

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