Biotechnology firm Alexion Pharmaceuticals has secured approval from the Ministry of Health, Labour and Welfare (MHLW) in Japan for the use of its Soliris (eculizumab) drug to treat pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS).
Japan had already approved Soliris for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), another severe and ultra-rare disease that impairs the production of blood cells.
The company intends to start initial treatment with Soliris for patients with aHUS in Japan in the fourth quarter of 2013.
aHUS is an ultra-rare chronic genetic disease which can gradually damage important organs that can lead to stroke, heart attack, kidney failure and death.
The morbidities and premature mortality in aHUS is caused by chronic uncontrolled activation of the complement system, which results in thromobotic microangiopathy (TMA).
The drug particularly targets uncontrolled complement activation and is approved for the suppression of TMA in patients with aHUS.
Alexion executive vice president and chief commercial officer David Hallal said the approval of Soliris for the treatment of aHUS by the Japanese government brings life-transforming hope to patients suffering from this disease.
"We will initiate our disease awareness and education programs as we work closely with the healthcare community to support rapid and accurate diagnosis of aHUS as well as better informed treatment decisions," Hallal said.
The US FDA, the European Medicines Agency (EMA) and other regulatory authorities have also approved Soliris for the treatment of patients with aHUS to inhibit complement-mediated TMA.
About 65% of all patients with aHUS die, while other will require kidney dialysis or have permanent kidney damage within the first year after diagnosis despite plasma exchange or plasma infusion (PE/PI).