Alexion Pharmaceuticals: Soliris Gains Another FDA Orphan Drug Designation
Alexion Pharmaceuticals’ orphan drug Soliris (Eculizumab) receives on June 13th, another FDA Orphan Drug Designation (ODD), for the treatment of Myasthenia Gravis (MG). 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.
MG is a rare neurological disorder caused by “uncontrolled complement activation”. This is a result of tissue damage and interference with signaling between nerve and muscle fibers in the nerve-muscle junction. At first, patients have eye muscle weakness and can progress to head, limb, respiratory, and spinal muscles. Currently, there is no cure for MG. Treatments include medications, surgical removal of the thymus gland, and plasma exchange. According to the NORD website, MG has a prevalence in the United States of approximately 14 – 40/100,000 people. According to an Orphanet Report, MG has a prevalence of approximately 20/100,000 people.
Multiple FDA Orphan Drug Designation Indications
Since 2003, Soliris receives FDA Orphan Drug Designation (ODD) for the following indications:
|Num||Designation Date/Approval Date||Indication|
|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|
FDA Breakthrough Therapy Designations
Alexion Pharmaceuticals is also developing other rare disease drug candidates to lessen the dependence on Soliris for revenue and growth. Two of these drug candidates receive the FDA Breakthrough Therapy Designation (BTD) in 2013 :
|2||Cyclic Pyranopterin Monophosphate (cPMP)||Molybdenum CofactorDeficiency(MoCD) Type A|
Please Note: “Soliris Product Packaging” courtesy of Alexion Pharmaceuticals Media Gallery.
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