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Rare Diseases And Orphan Drugs: Global News (09.19.13)

September 19, 2013

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

I – Japan

Alexion Pharmaceuticals announces September 13, 2013, that Japan’s Ministry of Health, Labor and Welfare (MHLW) approves orphan drug Soliris (Eculizumab) for the treatment of pediatric and adult patients with the ultra-rare disease, Atypical Hemolytic Uremic Syndrome (aHUS). The company expects to start treating the first aHUS patients with Soliris during the 4th Quarter of 2013.

Soliris is already approved in Japan for ultra-rare disease, Paroxysmal Nocturnal Hemoglobinuria (PNH). 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’ regulatory history :

•   Approved in 40 countries for PNH, a rare genetic blood disorder
•   Approved in US and EU for aHUS.

II – Europe

The European Commission (EC) last week approves for marketing in the European Union (EU), Raptor Pharmaceutical’s Procysbi (Cysteamine Bitartrate) as an orphan medicinal product for the rare disease Nephropathic Cystinosis. The approval of the Marketing Authorization Application (MAA) comes after the positive recommendation by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) in June 2013. The U.S. FDA approves Procysbi for the same indication in April 2013.  Nephropathic Cystinosis is a rare life-threatening metabolic Lysosomal Storage Disorder (LSD).

PTC Therapeutics announces that 2 Abstracts will be presented at the 10th European Pediatric Neurology Society Congress (EPNS), September 25-28, 2013, in Brussels, Belgium, on the following topics :

•   Confirmatory Phase III study of Ataluren in patients with nonsense mutation Duchenne Muscular Dystrophy (DMD)
•   Survey of patients with nonsense mutation DMD on symptoms and daily living activities.

Per the September 17th OrphaNews Europe Editorial, Germany has “elaborated and adopted the National Plan for Rare Disease, which will guide and structure actions in rare diseases within their health and social systems”. There are a total of 52 policy proposals in the National Plan.

The 7th European Conference on Rare Disease and Orphan Products (ECRD 2014) is scheduled for May 8 – 10, 2014, in Berlin, Germany. Posters are now being accepted for the meeting.

III – Australia

The Australian Government’s Therapeutic Goods Administration (TGA) has updated the list of designated orphan drugs as of September 19, 2013. Currently, there are 10 approved orphan drugs available to 7 different rare diseases which are funded by the Life Saving Drugs Program :

•   Gaucher Disease: Cerezyme (Imiglucerase), Vpriv (Velaglucerase Alfa), Zavesca (Miglustat)
•   Fabry Disease: Replagal (Agalsidase Alfa), Fabrazyme (Agalsidase Beta)
•   Mucopolysaccharidosis Type I (MPS I) : Aldurazyme (Laronidase)
•   Mucopolysaccharidosis Type II (MPS II) : Elaprase (Idursulfase)
•   Mucopolysaccharidosis Type VI (MPS VI) : Naglazyme (Galsulfase)
•   Infantile-Onset Pompe Disease : Myozyme (Alglucosidase Alfa)
•   Paroxysmal Nocturnal Haemoglobinuria (PNH) : Soliris (Eculizumab).

IV – United States

The Therapeutics for Rare and Neglected Diseases (TRND) program at the NIH’s (National Institutes of Health) National Center for Advancing Translational Sciences is funding the following 4 new preclinical drug development projects for treatments for rare diseases :

•   Hypoparathyroidism – 1st collaboration with a large pharmaceutical company, Eli Lilly
•   Hypertrophic cardiomyopathy associated with LEOPARD Syndrome
•   2 projects for Retinitis Pigmentosa, a severe form of hereditary blindness (1 project uses stem cells).

These projects represent the first time the program has :

•   A collaboration with a large pharmaceutical company
•   Used stem cells.

Please Note: “ApplicAtations-internet” PNG File By The Tango!Desktop Project | Wikimedia Commons.

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