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FDA Grants Breakthrough Therapy Designation To CPMP Replacement Therapy For Patients With Molybdenum Cofactor Deficiency (MoCD) Type A

About Molybdenum Cofactor Deficiency (MoCD) Type A

MoCD Type A is a severe, ultra-rare and genetic metabolic disease affecting newborns in which a genetic deficiency of cPMP results in the inability of the body to form an essential cofactor called molybdenum cofactor, resulting in its absence. This cofactor is essential for the appropriate functioning of several critical metabolic enzymes. A deficiency or absence of this cofactor leads to accumulation of toxic molecules, including the neurotoxin sulfite. Clinically, the absence of this cofactor and the resulting build-up of sulfite in the brain lead to damage and destruction of nerve cells, brain swelling, uncontrollable seizures, catastrophic and irreversible brain damage, and ultimately, death. 2 There are currently no treatment options for patients with MoCD Type A.

About cPMP/ALXN1101

ALXN1101 is a synthetic version of cPMP and is designed to replace the naturally occurring cPMP lacking in infants with MoCD Type A. ALXN1101 restores the deficient enzyme activity which then causes clearance of the toxic metabolite sulfite thereby preventing the irreversible neurologic damage observed in untreated patients with MoCD Type A. Encouraging early clinical experience with an earlier form of cPMP replacement therapy has been reported by independent investigators in Germany and Australia. 3

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on serving patients with severe and ultra-rare disorders through the innovation, development and commercialization of life-transforming therapeutic products. Alexion is the global leader in complement inhibition and has developed and markets Soliris ® (eculizumab) as a treatment for patients with PNH and aHUS, two debilitating, ultra-rare and life-threatening disorders caused by chronic uncontrolled complement activation. Soliris is currently approved in nearly 50 countries for the treatment of PNH, and in the United States, European Union, Japan and other countries for the treatment of aHUS. Alexion is evaluating other potential indications for Soliris in additional severe and ultra-rare disorders beyond PNH and aHUS, and is developing other highly innovative biotechnology product candidates across multiple therapeutic areas. This press release and further information about Alexion Pharmaceuticals, Inc. can be found at:


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