Human medicines European public assessment report (EPAR): Nulibry, fosdenopterin, Metal Metabolism, Inborn Errors, Date of authorisation: 15/09/2022, Revision: 2, Status: Authorised
Nulibry is a medicine used to treat patients with molybdenum cofactor deficiency (MoCD) type A.
MoCD type A is an inherited disease where patients do not have enough of a substance called 'molybdenum cofactor', a molecule needed for the production of certain enzymes. Without these enzymes, the toxic chemical sulfite builds up in the brain causing brain damage.
In the 'type A' form of the disease, the absence of molybdenum cofactor is due to patients lacking a substance called 'cyclic pyranopterin monophosphate (cPMP)’, which the body needs to make molybdenum cofactor.
MoCD type A is rare, and Nulibry was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 September 2010. Further information on the orphan designation can be found here.
Nulibry contains the active substance fosdenopterin.
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