Alexion’s drug Soliris (eculizumab) has received marketing approval from Health Canada for the treatment of patients with the ultra-rare and life-threatening genetic condition, atypical haemolytic uraemic syndrome (aHUS).
aHUS causes abnormal clots to form in the blood vessels throughout the body, leading to progressive and catastrophic damage to the vital organs, including the kidneys, brain and heart. The pathogenesis of this abnormal clotting – or thrombotic microangiopathy (TMA) – is the chronic uncontrolled activation of the complement system.
Soliris is the first and only treatment for aHUS. It is a recombinant fully-humanized hybrid IgG2/IgG4 monoclonal antibody that blocks the terminal portion of the complement cascade, specifically targeting complement component 5.
Previous management of aHUS relies on plasma infusion and plasma exchange therapies that not only have variable results but also can be costly, painful and time-consuming.
“Soliris has been shown to have a life-changing impact on patients with aHUS and represents a major advance in the treatment of this extremely rare but devastating disease that previously had no treatment options,” said Anne-Laure Lapeyraque, M.D., MSc., Assistant Professor of Paediatrics, Division of Nephrology, at the Hospital Sainte Justine, Université de Montréal. “By directly targeting uncontrolled complement activation, the underlying cause of the progressive organ failure and shortened lifespan for patients with aHUS, Soliris can markedly decrease the TMA process, improve kidney function, and ultimately has the potential to change the course of aHUS.”
Developed by Alexion Pharmaceuticals, Soliris has already received approval in the United States and the European Union for treatment of patients with aHUS. The drug is also approved in the US, Canada, the EU and Japan for patients with paroxysmal nocturnal haemoglobinuria (PNH). Health Canada has issued marketing authorization for adolescents and adults (aged 13-17) with atypical HUS to reduce complement-mediated TMA.
Do you think eculizumab is on the way to becoming the new standard of care for aHUS?
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If you’d like to hear more about orphan drug development, attend the 4th World Orphan Drug Congress Europe, in Geneva on the 14th November 2013.