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cyclic pyranopterin monophosphate (cPMP) replacement therapy (ALXN1101)

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Company Alexion Pharmaceuticals Inc.
DescriptionIntravenous Escherichia coli-derived cyclic pyranopterin monophosphate (cPMP)
Molecular Target
Mechanism of Action 
Therapeutic ModalitySmall molecule
Latest Stage of DevelopmentPhase II
Standard Indication Neurology (unspecified)
Indication DetailsTreat molybdenum cofactor deficiency (MoCD) type A
Regulatory Designation

U.S. - Breakthrough Therapy (Treat molybdenum cofactor deficiency (MoCD) type A)

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