Alexion Recruiting Patients in Four Soliris Clinical Studies in
Europe, United States and Canada
Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced today that Soliris®
(eculizumab), its first-in-class complement inhibitor, has been granted
Orphan Medicinal Product Designation by the European Commission for the
treatment of patients with atypical Hemolytic Uremic Syndrome (aHUS).
AHUS is an ultra-rare, inherited, and life-threatening
complement-inhibitor deficiency disease that often progresses to
end-stage kidney disease or failure. The U.S. Food and Drug
Administration granted orphan drug designation to Soliris for the same
indication in May 2009. Soliris is not approved for the treatment of
patients with aHUS.
Alexion is currently enrolling patients at the initial sites in four
clinical studies of Soliris as an investigational treatment for
adolescent and adult patients with aHUS. Clinical studies are also
currently being planned to investigate the use of Soliris as a treatment
for children with aHUS. If Soliris is approved for the treatment of
patients with aHUS in Europe or the U.S., orphan-drug status would
entitle Alexion to 10 years of market exclusivity in Europe and seven
years of market exclusivity in the U.S. for this use of Soliris.
Soliris is approved in the United States, European Union, Australia and
Canada as a treatment for patients with paroxysmal nocturnal
hemoglobinuria (PNH), an ultra-rare, debilitating, and life-threatening
blood disorder. Soliris has also been designated as an orphan drug in
these countries, as well as in Japan, for the treatment of patients with
PNH.
"These additional orphan drug designations for Soliris underscore the
unmet need faced by patients living with aHUS, an ultra-rare, genetic
and life-threatening disease that destroys patients’ kidneys,” said
Leonard Bell, M.D., Chief Executive Officer of Alexion. “In response to
the work of researchers and inquiries from practicing physicians, we are
increasingly focusing our resources to investigate Soliris as a
treatment for patients with aHUS.”
About the aHUS Clinical Studies
Atypical Hemolytic Uremic Syndrome is characterized by chronic
inflammation, hemolysis (red blood cell destruction), thrombocytopenia
(reduced circulating platelets), and microangiopathy (damage in small
blood vessels), particularly in the kidney and brain, often progressing
to end-stage kidney disease or failure. Like PNH, aHUS is caused by a
deficiency in normally occurring complement inhibitor proteins.
Typically, patients with aHUS have genetic mutations in one of several
complement inhibitor proteins that lead to uncontrolled complement
activation. Excessive complement activation may contribute to severe
inflammation of the blood vessels and blood clotting through the
activation of white blood cells, platelets, and the endothelial cell
lining of blood vessels. (1)
The prognosis for patients with aHUS is generally poor. Approximately 70
percent of patients with the most common mutation experience chronic
renal insufficiency, chronic dialysis, or death within one year of the
first clinical episode.
