Life-Threatening Blood Disorder Treatment Gets Orphan Drug Status

PNH is a debilitating, life-threatening blood disorder in which can lead to hemolysis
PNH is a debilitating, life-threatening blood disorder in which can lead to hemolysis

Alexion has announced that the Food and Drug Administration (FDA) has granted orphan drug designation to ALXN1210, their candidate treatment for paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a debilitating, ultra-rare, life-threatening blood disorder in which uncontrolled activation of complement results in hemolysis. ALXN1210 is a longer-acting anti-C5 antibody that inhibits terminal complement. Results from early studies have shown promising reduction of free C5 levels. 

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“Data from our ongoing clinical studies have shown rapid, complete, and sustained complement inhibition in treated patients,” said Martin Macky, PhD and EVP at Alexion. “We look forward to continuing to evaluate this highly innovative molecule in our Phase 3 trial of ALXN1210.”

Phase 3 trials of ALXN1210 (ID #'s NCT02946463 and NCT02949128) in patients with PNH and atypical hemolytic uremic syndrome (aHUS), another life-threatening disease caused by chronic uncontrolled complement activation, are currently underway.

For more information visit Alexion.com.