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Alexion Pharmaceuticals: Soliris Gains Another FDA Orphan Drug Designation

June 14, 2014


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.

Myasthenia Gravis

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.

June is National Myasthenia Gravis Awareness Month.

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
1 08-20-2003/ 03.16.2007 PNH
2 04-29-2009/ 09.23.2011 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
6 06-13-2014 Myasthenia Gravis


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 :

Num Drug Name Indication
1 Asfotase Alfa Hypophosphatasia
2 Cyclic Pyranopterin Monophosphate (cPMP) Molybdenum CofactorDeficiency(MoCD) Type A



European Myasthenia Gravis Association (EuMGA)

Myasthenia Gravis (MG) Association

Myasthenia Gravis Foundation of America (MGFA).

Please Note: “Soliris Product Packaging” courtesy of Alexion Pharmaceuticals Media Gallery.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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