What has SMC said?
Fosdenopterin was assessed by the Scottish Medicines Consortium (SMC) and meets the definition of an ultra-orphan medicine, which is a medicine to treat an extremely rare condition.
This document summarises the initial SMC assessment of fosdenopterin for treating patients with molybdenum cofactor deficiency (MoCD) Type A, a rare inherited metabolic condition where the body does not produce enough of a substance called molybdenum cofactor. This leads to a build-up of sulphite in the brain which causes brain damage.
You can find more detailed information by looking at the SMC ultra-orphan initial assessment (SMC2624).
What does SMC’s assessment mean for patients?
The Scottish Government will confirm when fosdenopterin is available for prescribing on the NHS in Scotland as part of the ultra-orphan pathway.
Your healthcare professional can discuss with you if it is the right treatment for you or your child.
You can find more information about making decisions about your or your child’s treatment in this booklet called: Medicines in Scotland: What’s the right treatment for me?
What has SMC said in this assessment?
- A combined analysis of five different clinical studies suggested that fosdenopterin is associated with benefits such as improving overall survival and increasing the time to when patients can no longer feed orally.
- There were limits to the clinical evidence as the studies had small sample sizes and were types that are prone to bias. Therefore, there is uncertainty in the size of the benefits of fosdenopterin.
- If given early enough fosdenopterin may offer the potential for patients to take part in daily activities and improve their quality of life.
- Despite the confidential discount offered by the company, the cost in relation to the health benefits of fosdenopterin remains high.
How does SMC assess ultra-orphan medicines?
SMC uses a broad assessment framework for ultra-orphan medicines. This is part of the ultra-orphan pathway in NHSScotland.
You can find out more about how SMC assesses medicines in the ultra-orphan pathway here: https://www.scottishmedicines.org.uk/how-we-decide/ultra-orphan-medicines-for-extremely-rare-conditions
More information and support
The organisation below can provide more information and support for people with MoCD and their families. SMC is not responsible for the content of any information provided by external organisations.
Metabolic Support UK
https://metabolicsupportuk.org
0800 652 3181
You can find out more about fosdenopterin (Nulibry®) in the Patient Leaflet by searching for the medicine name on the Medicines and Healthcare products Regulatory Agency (MHRA) website.