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Fosdenopterin for treating molybdenum cofactor deficiency type A

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1 Recommendations

1.1

Fosdenopterin is not recommended, within its marketing authorisation, for treating molybdenum cofactor deficiency (MoCD) type A in people of all ages.

1.2

This recommendation is not intended to affect treatment with fosdenopterin that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers.

Why the committee made these recommendations

MoCD type A is a rare genetic condition that can appear shortly after birth. It causes the build-up of sulfites in the body and the brain, which leads to seizures, feeding difficulty, loss of muscle control and poor survival. Standard care includes treatments that manage symptoms, such as antiseizure medicines.

Clinical trial evidence suggests that fosdenopterin increases the time before people are unable to eat and how long they live compared with standard care. But the trials were very small and did not compare fosdenopterin with other treatments, so the results are highly uncertain.

There are also uncertainties in the economic model, including that it:

  • only captures evidence for 1 person with late-onset MoCD type A treated with fosdenopterin; most of the evidence was from people with early-onset MoCD type A

  • does not fully capture all the outcomes relevant to MoCD type A

  • does not include quality-of-life data from people with MoCD type A

Even when considering the condition's severity, its effect on quality and length of life, and the size of benefit with fosdenopterin, the cost-effectiveness estimates are substantially higher than what NICE considers an acceptable use of NHS resources for highly specialised technologies. So, fosdenopterin is not recommended.