Alexion Pharmaceuticals has announced that the European Commission has granted orphan drug designation (ODD) to ALXN1210, a highly innovative, longer-acting C5 antibody being evaluated in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a debilitating, ultra-rare, life-threatening blood disorder in which uncontrolled activation of complement, a component of the immune system, results in hemolysis (destruction of a patient’s red blood cells).
“Patients with PNH are at continuous risk for severe and life-threatening consequences resulting from the ongoing complement-mediated destruction of red blood cells,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “Soliris has been approved for the treatment of patients with PNH since 2007 and has dramatically changed the outlook for patients with this disease. Preliminary results from our ongoing clinical studies of ALXN1210, which has a half-life nearly three times that of Soliris, have shown rapid, complete, and sustained complement inhibition in treated patients with PNH. We are pleased that the Committee for Orphan Medicinal Products recognized that ALXN1210 has the potential to offer a meaningful clinical advantage for patients living with this devastating ultra-rare disorder.”
Alexion is evaluating ALXN1210 in a Phase 1/2 study and a Phase 2 study in patients with PNH. Both studies have exceeded target enrollment. More information on these clinical trials is available at www.clinicaltrials.gov under the identifiers NCT02598583 and NCT02605993. Alexion also plans to initiate a clinical program with ALXN1210 in patients with atypical hemolytic uremic syndrome (aHUS), another ultra-rare and life-threatening disease caused by chronic uncontrolled complement activation, in 2016. ALXN1210 is not approved in any country.
The European Commission grants orphan medicinal product status to provide incentives to develop medicinal products to treat, prevent or diagnose diseases or conditions that affect no more than five in 10,000 persons in the EU. The orphan medicinal product status designation provides Alexion with certain benefits and incentives in the EU, including a period of market exclusivity if ALXN1210 is approved to treat patients with PNH.