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Alexion Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to ALXN1210, a highly innovative, longer-acting anti-C5 antibody that inhibits terminal complement, which is being evaluated for the treatment of 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).
“Alexion is committed to achieving the highest levels of innovation to address the needs of patients suffering from PNH, a devastating ultra-rare disorder,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. “We are pleased that the FDA has recognized the potential for ALXN1210 to offer a significant therapeutic advantage for patients with PNH. Data from our ongoing clinical studies have shown rapid, complete, and sustained complement inhibition in treated patients, and we look forward to continuing to evaluate this highly innovative molecule in our Phase 3 trial of ALXN1210 administered every eight weeks.”
Alexion is currently enrolling patients in Phase 3 trials of ALXN1210 in patients with PNH as well as in patients with atypical hemolytic uremic syndrome (aHUS), another ultra-rare and life-threatening disease caused by chronic uncontrolled complement activation. In June 2016, ALXN1210 was granted ODD by the European Commission for the treatment of patients with PNH. ALXN1210 is protected by a composition of matter patent in the U.S. and Europe through 2035. ALXN1210 is not approved in any country.
PNH is an ultra-rare blood disorder in which chronic, uncontrolled activation of complement, a component of the normal immune system, results in hemolysis (destruction of the patient's red blood cells). PNH strikes people of all ages, with an average age of onset in the early 30s.
ALXN1210 is a highly innovative, longer-acting anti-C5 antibody discovered and developed by Alexion that inhibits terminal complement. In early studies, ALXN1210 demonstrated rapid, complete, and sustained reduction of free C5 levels. Alexion has completed enrollment in two ongoing clinical studies of ALXN1210 in patients with PNH—a Phase 1/2 dose-escalating study and an open-label, multi-dose Phase 2 study that is also evaluating longer dosing intervals beyond 8 weeks.
ALXN1210 is currently in Phase 3 trials in patients with PNH and aHUS. In addition, Alexion is conducting a Phase 1 study to evaluate a new formulation of ALXN1210 administered subcutaneously in healthy volunteers. In June 2016, the European Commission granted Orphan Drug Designation (ODD) to ALXN1210 for the treatment of patients with PNH
