Alexion Pharmaceuticals, Inc
) 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
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.
Anika Therapeutics Inc.
Jazz Pharmaceuticals Public Limited Company
) appear to be more favorably placed with a Zacks Rank #1 (Strong
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