Alxion Pharma receives FDA orphan drug designation for PNH

Release Date: 12-Jan-2017

Alexion Pharmaceuticals Inc. has recently announced that US Food and Drug Administration has granted orphan drug status to leading candidate ALXN 1210 for the treatment of patients suffering from 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 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.


Orphan Drug Designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.



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