Alexion Pharmaceuticals: Soliris Continues To Reach New Heights
Alexion Pharmaceuticals, a Connecticut-based global biotechnology company developing treatments for ultra-rare diseases, reports on October 23rd the company’s 3rd quarter 2014 results. 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.
I – 3rd Quarter 2014 Results Summary
Here are some of the highlights from Alexion Pharmaceuticals’ 3rd quarter 2014 results:
• 3rd quarter 2014 Soliris net product sales increases approximately 38.6% to $555.1 million from 3rd quarter 2013 net product sales of $400.4 million
• Global Soliris growth continues for PNH & aHUS indications in close to 50 countries
• Juvenile-Onset Hypophosphatasia (HPP) natural history study completed for Asfotase Alfa
• Asfotase Alfa FDA rolling Biologics License Application (BLA) submission scheduled to be completed in 4th quarter 2014
• New Drug Application (NDA) submission for Asfotase Alfa in Japan in October 2014
• Clinical development of the first 2 molecules in the Company’s innovative portfolio of next-generation Soliris candidates started.
Leonard Bell, M.D., Chairman and CEO of Alexion Pharmaceuticals, says:
“ … Our third quarter performance underscores the significant opportunity we have to serve more patients with PNH and aHUS globally. While we remain focused on our initiatives to reach more patients in our current operations, we are also preparing for the global launch of asfotase alfa in 2015 and advancing our lead development programs as we drive toward as many as seven additional launches through 2018.””
Alexion Pharmaceuticals continues to expand Soliris’ franchise and adds to the company’s orphan drug pipeline, which results in the increase in income and the steady penetration into more than 40 global markets.
II – 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|
III – FDA Breakthrough Therapy Designations (BTDs)
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|
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