Alexion receives orphan drug designation for PNH treatment

PNH leads to hemolysis, in which a person’s red blood cells are destroyed. | File photo

The U.S. Food and Drug Administration granted orphan drug designation to Alexion Pharmaceuticals for ALXN1210, a drug for treating patients with paroxysmal nocturnal hemoglobinuria (PNH).

PNH, a life-threatening blood disorder, is very rare in the United States. The disorder involves the uncontrolled activation of complement, a component of the immune system. This issue leads to hemolysis, in which a person’s red blood cells are destroyed.

“Alexion is committed to achieving the highest levels of innovation to address the needs of patients suffering from PNH, a devastating ultra-rare disorder,” Dr. Martin Mackay, executive vice president and global head of R&D at Alexion, said in a statement. “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.”

Through its Office of Orphan Products Development, the FDA grants special status to effective treatment options for diseases or disorders that affect fewer than 200,000 people in the United States and provides the drug’s maker with certain benefits and incentives.

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Organizations in this story

Alexion Pharmaceuticals, Inc. 100 College St New Haven, CT 06510