Good news flowed in at Alexion Pharmaceuticals, Inc. (ALXN) from Japan when the country’s Ministry of Health, Labour and Welfare approved the company’s sole marketed product, Soliris (eculizumab), for treating patients suffering from atypical hemolytic uremic syndrome (aHUS). We note that aHUS is an ultra-rare chronic genetic disease that can prove to be fatal in certain cases.
Alexion had filed for approval of Soliris for treating aHUS on the basis of encouraging data from five studies. Two of the studies evaluated adults and pediatrics suffering from aHUS and another (a retrospective study) studied Soliris on aHUS children. Japanese approval was also sought on the basis of data from an ongoing prospective study in addition to a retrospective study on Soliris in patients (adults and pediatrics) suffering from aHUS. Alexion expects to launch Soliris for treating aHUS patients in Japan by year end.
Apart from Japan, Soliris is approved for treating aHUS in the U.S., EU and many other countries. The U.S. and EU approvals for the indication came in 2011. The Japanese approval of Soliris for treating aHUS patients will boost the drug’s sales potential further. The drug recorded sales of $1.13 billion in 2012, up 45%.
Soliris is also available in multiple countries, including Japan, for treating patients suffering from paroxysmal nocturnal hemoglobinuria (:PNH). PNH is a rare genetic blood disorder characterized by the onset of severe hemolytic anemia, chronic fatigue and intermittent episodes of dark urine, known as hemoglobinuria.
Alexion is working on expanding Soliris’ label into additional indications like Shiga-toxin producing E. coli hemolytic uremic syndrome and myasthenia gravis. Alexion’s pipeline also includes asfotase alfa, ALXN1102/ALXN1103 and ALXN1007.
Alexion currently carries Zacks Rank #3 (Hold). Stocks which appear to be more attractive, include Gilead Sciences Inc. (GILD), Actelion Ltd. (ALIOF) and Biogen Idec Inc. (BIIB), each with a Zacks Rank #1 (Strong Buy).