Alexion Pharmaceuticals, Inc. (ALXN - Free Report) recently announced that encouraging data on its sole marketed drug Soliris has been published in the New England Journal of Medicine (NEJM) for treating patients suffering from atypical hemolytic uremic syndrome (aHUS).
Data from the two pivotal studies published in the NEJM revealed that treatment with Soliris significantly inhibits systemic complement-mediated thrombotic microangiopathy (TMA) and reduces the need for TMA-related intervention. Moreover, chronic Soliris therapy also results in improvement in platelet count as well as renal function in aHUS patients. Moreover, treatment with Soliris leads to the reversal of vital organ damage and causes improvements in the quality of life (related to health). The study also indicated that earlier intervention with Soliris caused clinical outcomes to improve.
We note that Soliris is available in the US, EU and many other countries for the treatment of paroxysmal nocturnal hemoglobinuria, a rare genetic blood disorder. In Sep 2011, the US Food and Drug Administration (FDA) cleared Soliris for treating children and adults suffering from aHUS, an ultra-rare genetic disorder. Soliris was subsequently launched in the country. In Nov 2011, a similar approval for the drug was granted in the EU.
Alexion Pharma is working on developing its pipeline to reduce its dependence on Soliris for growth. The company is also working on expanding Soliris’ label into additional indications like Shiga-toxin producing e. coli hemolytic uremic syndrome, neuromyelitis optica and myasthenia gravis. Alexion Pharma’s pipeline also includes asfotase alfa (hypophosphatasia), ALXN1102/ALXN1103 (hematology), and ALXN1007 (inflammatory disorders).
Last month, the FDA granted Breakthrough Therapy designation, which forms a part of the FDA Safety and Innovation Act of 2012, to asfotase alfa for treating patients suffering from hypophosphatasia. The symptoms were witnessed in the patients prior to attaining adulthood. Alexion Pharma stated in its press release that the status is assigned to speed up the development of the drug for treating a serious or life-threatening disorder.
Alexion Pharma carries a Zacks Rank #2 (Buy). Companies such as Biogen Idec Inc. (BIIB - Free Report) , Anika Therapeutics Inc. (ANIK - Free Report) and Jazz Pharmaceuticals Public Limited Company (JAZZ - Free Report) appear to be more favorably placed with a Zacks Rank #1 (Strong Buy).