The FDA has granted orphan drug designation to ALXN1210, a highly innovative, longer-acting anti-C5 antibody from Alexion Pharmaceuticals 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).

“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,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion.

more