Soliris® in Other Diseases
Alexion's research and development pipeline is now focused on investigating the use
of Soliris to block complement-mediated inflammation in patients with other rare
and severe conditions, including chronic blood cell and clotting disorders, kidney
diseases and transplant rejection, and chronic and debilitating neurological disorders.
This focus is supported by a November 2007 article28 in the journal
Nature Biotechnology, which noted that careful, rigorous scientific evaluation
similar to that used in the development of Soliris in PNH may be applied to the
evaluation of this first-in-class complement inhibitor in patients with other uncommon,
debilitating and life-threatening diseases.
In
kidney disorders, Alexion is initiating clinical trials to evaluate Soliris
for the treatment for patients with atypical Hemolytic Uremic Syndrome, or
aHUS, a rare disease that can lead to kidney failure or death. Independent investigators
have started a study to evaluate Soliris in patients at high risk for kidney transplant rejection. Independent
physicians are also evaluating the use of Soliris in patients with Dense Deposit Disease (DDD).
In hematology,
Alexion is planning studies of Soliris in patients with Catastrophic Anti-Phospholipid Syndrome
(CAPS) and
Cold Agglutinin Disease (CAD), two rare blood cell disorders.
In severe
neurologic disorders, the first patients with Myasthenia Gravis, a neuromuscular disease, are
now being treated with Soliris in a double-blind placebo-controlled trial. Independent
investigators have also started to enroll patients in a study to evaluate Soliris
for the treatment of
Mutlifocal Motor Neuropathy (MMN).