Alexion Announces FDA Breakthrough Therapy Designation for cPMP Replacement Therapy for Patients with MoCD Type A

Alexion Pharma International Sàrl, a subsidiary of Alexion Pharmaceuticals, Inc.

, today announced that the U.S. Food and Drug Administration (FDA) has granted a Breakthrough Therapy designation to cyclic pyranopterin monophosphate (cPMP, or ALXN1101), an enzyme co-factor replacement therapy for the treatment of patients with molybdenum cofactor deficiency (MoCD) type A, a severe and life-threatening, ultra-rare, genetic metabolic disorder that causes catastrophic and irreversible neurologic damage within the first weeks of life. According to the FDA, a Breakthrough Therapy designation is designed to expedite the development of a drug to treat a serious or life-threatening disease when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement