European Commission Grants Orphan Medicinal Product Designation To Soliris

European Commission has granted Orphan Medicinal Product Designation to Alexion’s Soliris (eculizumab), for the treatment of patients with atypical Hemolytic Uremic Syndrome (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.

Reportedly, clinical studies are also being planned to investigate the use of Soliris as a treatment for children with aHUS.

Moreover, if Soliris is approved for the treatment of patients with aHUS in Europe or the US, orphan-drug status would entitle Alexion to ten years of market exclusivity in Europe and seven years in the US for the use of Soliris.

Soliris is approved in the US, EU, 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.

Leonard Bell, CEO of Alexion, said: 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.

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, he added.