Alexion Says FDA Grants Orphan Drug Designation To ALXN1210 For Treatment Of PNH

(RTTNews) - Alexion Pharmaceuticals Inc. (ALXN) said that the U.S. Food and Drug Administration 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 or 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 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.