Fosdenopterin for molybdenum cofactor
deficiency type A


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Fosdenopterin is currently in clinical development for the treatment of paediatric patients aged 1 day to 5 years with molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD type A cannot produce a substance known as cyclic pyranopterin monophosphate (cPMP).

Interventions: Fosdenopterin
Year: 2023

Fosdenopterin is currently in clinical development for the treatment of paediatric patients aged 1 day to 5 years with molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD type A cannot produce a substance known as cyclic pyranopterin monophosphate (cPMP). MoCD type A is a rare genetic disease that can appear shortly after birth and is caused by the mutation of the MOCS1 gene in the body. This leads to the deficiency of certain compounds and enzymes such as sulfite oxidase (SOX) and without these enzymes, the toxic chemical sulfite can build-up in the brain. This leads to symptoms such as seizures (fits), severe brain abnormalities, bleeding in the brain, increased reflex responses and feeding problems, alongside the severity and poor survival associated with the condition. Patients with MoCD type A who survive beyond infancy typically suffer from progressive brain damage, and an inability to make coordinated movements or communicate with their environment. Currently there are no licensed or recommended therapies available for patients with MoCD in the UK.