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Alexion Pharmaceuticals: The Power Of Soliris Continues

January 17, 2014

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:


Designation Date




08-20-2003 PNH


04-29-2009 aHUS


10-18-2011 Shiga-Toxin producing Escherichia Coli Hemolytic Uremic   Syndrome (STEC-HUS)


06-24-2013 NeuroMyelitis Optica (NMO)


01-10-2014 Prevention of Delayed Graft Function (DGF)  after Renal Transplantation


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, in San Francisco. Here are a few highlights from the presentation :

•   Exclusive focus on only creating, developing, and delivering therapies for ultra-rare diseases

•   Current commercial operations are largely focused on Soliris

•   PNH is the 1st approved indication for Soliris & now serves approximately 50 countries

•   aHUS is the 2nd approved indication for Soliris, which is on track to equal or exceed the performance to serve patients in PNH

•   Expect company’s 2nd rare disease product, orphan drug Asfotase Alfa, for rare disease Hypophosphatasia (HPP), to launch later this year

•   2014 – 2018, several rare disease treatment launches now positioned for :

  1. Asfotase Alfa for HPP at end of 2014
  2. Solaris for 2 kidney transplant indications: Antibody Mediated Rejection (AMR) & Delayed Graft Function (DGF)
  3. Soliris for 2 neurology indications: Neuromyelitis Optica (NMO) & Myasthenia Gravis (MG)
  4. Orphan Drug cPMP Replacement Therapy for a lethal newborn disorder, Molybdenum Cofactor Deficiency Type A (MoCD)

•   Asfotase Alfa, for HPP, is the 1st non-Solaris breakthrough candidate :

  1. HPP is rare hereditary metabolic disorder caused by a protein deficiency, which causes patients to not  make effective bone
  2. Over ½ of newborns born with this disorder will not reach their 1st birthday
  3. Rreceives FDA Breakthrough Therapy Designation in 2013
  4. Plan to file marketing applications in 2014 in US, Europe, & Japan

•   Focus next on cPMP Replacement Therapy for MoCD Type A, which receives FDA Breakthrough Therapy Designation in 2013

•   Agreement this week with Moderna Therapeutics to develop mRNA therapeutics for rare diseases.

Continuation Of Slow, Steady, Global Growth Of Soliris

Alexion Pharmaceuticals will continue a slow, steady, successful global growth from Soliris, by expanding the franchise :

•   Adding other indications

•   Expanding its geographic presence and penetration.

FDA Breakthrough Therapy Designations

Alexion Pharmaceuticals’ strategy is also to develop 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 :


Drug Name



Asfotase Alfa Hypophosphatasia


Cyclic                   Pyranopterin Monophosphate (cPMP) Molybdenum CofactorDeficiency(MoCD) Type A


An Agreement With Moderna Therapeutics

Alexion Pharmaceuticals is also reducing its reliance on Soliris by signing an agreement on January 13, with Moderna Therapeutics for the discovery and development of messenger RNA (mRNA) therapeutics for rare diseases :

“Moderna is pioneering messenger RNA therapeutics, an entirely new in vivo drug modality that produces human proteins or antibodies inside patient cells, which are in turn secreted or active intracellularly.”

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

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

  1. Very inspiring! Look forward to getting updates on your success in WODC USA 2014!

    Be interesting to hear from other orphan drug developments – do they all apply to multiple indications, rare and/or common?

    See you then!

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