Alexion Pharmaceuticals is a global biopharmaceutical company focusing on developing therapies for patients with ultra-rare diseases. The company’s first and only marketed product, orphan drug Soliris (Eculizumab), generates blockbuster profits from two approved indications :
• Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare genetic blood disorder
• Atypical Hemolytic Uremic Syndrome (aHUS), an ultra-rare genetic disorder.
Multiple FDA Orphan Drug Designation Indications
Soliris has FDA Orphan Drug Designation (ODD) for the following indications:
Num | Designation Date | Orphan Designation |
1 | 08-20-2003 | PNH |
2 | 04-29-2009 | aHUS |
3 | 10-18-2011 | Shiga-Toxin producing Escherichia Coli Hemolytic Uremic Syndrome (STEC-HUS) |
4 | 06-24-2013 | NeuroMyelitis Optica (NMO) |
5 | 01-10-2014 | Prevention of Delayed Graft Function (DGF) after Renal Transplantation |
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On January 10, Soliris receives FDA ODD for the prevention of Delayed Graft Function (DGF) after renal transplantation.
J.P. Morgan Healthcare Conference
Leonard Bell, CEO of Alexion Pharmaceuticals, presents on January 15 at the J.P. Morgan Healthcare Conference
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DRUG APPROVALS BY DR ANTHONY MELVIN CRASTO
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