SMITHFIELD – Alexion Pharmaceuticals Inc. has finished enrolling patients in four open-label clinical trials of its drug Soliris, the company’s only product.
The Cheshire, Conn.-based pharmaceutical company, which operates a manufacturing plant in Smithfield, said last week it has achieved its enrollment targets for clinical studies being run in North America and Europe.
The company is investigating whether Soliris (eculizumab) could be used to treat patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare genetic disorder characterized by sudden clinical deterioration. The outcome for patients diagnosed with aHUS is often poor. Alexion hopes Soliris may provide another treatment option for aHUS patients, particularly for those who are resistant to plasma therapy.
“Given the potential for rapid and life-threatening deterioration observed in patients with aHUS, we are increasingly focused on diligently advancing our developmental efforts,” said Alexion CEO Dr. Leonard Bell. “In addition, because a significant number of patients with aHUS are children, we are committed to expanding our aHUS program to include studies of Soliris in pediatric patients with aHUS.”
Alexion plans to present preliminary results of the studies later this year, according to Bell.
Alexion is currently working with regulators in the United States and the European Union to establish protocols for conducting studies of Soliris in patients younger than age 12.
Soliris is approved in the U.S. and European Union to treat patients with paroxysmal nocturnal hemoglobinuria, an ultra-rare blood disorder characterized by the destruction of red blood cells.
atypical Hemolytic Uremic Syndrome,
Alexion CEO Dr. Leonard Bell,
paroxysmal nocturnal hemoglobinuria