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Alexion Pharmaceuticals Receives 15th FDA Breakthrough Therapy Designation

May 28, 2013

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Alexion Pharmaceuticals, a Cheshire, Connecticut, biopharmaceutical company developing therapies for ultra-rare diseases, announces on May 28, 2013, that their subsidiary Alexion Pharma International Sarl’s orphan drug Asfotase Alfa  receives FDA Breakthrough Therapy Designation #15. Asfotase Alfa is for the treatment of patients with Hypophosphatasia (HPP) whose first signs or symptoms occurred prior to 18 years of age. The HPP indication includes perinatal-, infantile-, and juvenile-onset forms of the disease. The FDA also confirms that adult-onset HPP is “a serious and life threatening disease or condition”, and that Breakthrough Therapy Designation could be obtained for this aspect of the disease with additional clinical information.

HPP is an inherited, life-threatening, ultra-rare metabolic disorder that leads to progressive damage to multiple organs, including destruction and deformity of bones. HPP is caused by a genetic deficiency of an enzyme known as Tissue Non-Specific Alkaline Phosphatase (TNSALP), which causes abnormalities in metabolism of phosphate and calcium, resulting in mortalities.

Asfotase Alfa is an investigational first-in-class targeted Enzyme Replacement Therapy (ERT). Asfotase Alfa receives FDA Orphan Drug Designation (ODD) in September 2008 and also FDA’s Fast Track status. Asfotase Alfa works by “normalizing the genetically defective metabolic process, and preventing or reversing the severe and life-threatening complications of life-long dysregulated mineral metabolism.”

Alexion Pharmaceuticals’ orphan drug Soliris (Eculizumab), the world’s most expensive drug, is a treatment for 2 ultra-rare diseases :

•   Paroxysmal Nocturnal Hemoglobinuria (PNH)
•   Atypical Hemolytic Uremic Syndrome (aHUS).

Soliris is currently approved in more than 35 countries for the treatment of PNH, and in the United States and the European Union (EU) for the treatment of aHUS.

Please Note: FDA Official Logo from FDA website.

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