NICE supports the commission of Soliris for aHUS

• Britain's National Institute for Health and Care Excellence (NICE), the advisory group for the National Health Service, recommends the commission of Alexion Pharmaceuticals' (NASDAQ:ALXN) Soliris (eculizumab) for the treatment of patients with atypical hemolytic uremic syndrome (aHUS), an ultra-rare life-threatening disorder characterized by renal failure and the formation of blood clots in the small blood vessels throughout the body (thrombotic microangiopathy or TMA). In the UK, the total number of potential patients, typically children five years old or less, is only ~25.
• 79% of patients with aHUS die within three years of diagnosis despite plasma exchange or plasma infusion. The majority of patients with aHUS who undergo a kidney transplant experience subsequent systemic TMA and face a 90% transplant failure rate.
• Soliris first achieved regulatory clearance in 2007 in the U.S. and EU for the treatment of another ultra-rare life-threatening blood disorder called paroxysmal nocturnal hemoglobinuria (PNH). It was approved for aHUS in the U.S. and EU in 2011. Soliris is also cleared for sale for both indications in Japan and selected other countries.
• A full regimen of Soliris costs ~$540,000, the world's most expensive drug until UniQure's Glybera (alipogene tiparvovec) leapfrogged it.
• NICE recommended funding for Soliris for aHUS in September.
• Previously: NICE recommends Soliris for aHUS
• Previously: UniQure sets price record with Glybera