Assessment of triheptanoin for treating long-chain fatty acid oxidation disorders

2025

Metabolic Support UK would like to keep you updated about the latest developments in the assessment of triheptanoin, also known as Dojolvi. This medicine is currently being assessed by the National Institute for Health and Care Excellence (NICE) in England.

Click the button below for our update:

Assessment of pegzilarginase for treating arginase-1 deficiency

2025

Metabolic Support UK would like to keep you updated about the latest developments in the assessment of pegzilarginase, also known as Loargys. This medicine is currently being assessed by the National Institute for Health and Care Excellence (NICE) in England. 

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Assessment of fosdenopterin for treating molybdenum cofactor deficiency type A

2025

Metabolic Support UK would like to keep you updated about the latest developments in the assessment of fosdenopterin, also known as Nulibry or cPMP. This medicine is currently being assessed by the National Institute for Health and Care Excellence (NICE) in England and the Scottish Medicines Consortium (SMC) in Scotland.

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Final Draft Guidance Recommends Asfotase alfa (Strensiq) for use for the treatment of Paediatric-onset HPP

1st February 2023

Metabolic Support UK are delighted to share that following a second committee meeting, NICE have now released final draft guidance to recommend the use of Asfotase alfa (Strensiq) for the treatment of all patients with perinatal and infantile forms of paediatric-onset hypophosphatasia and additional recommendations for those with juvenile forms who meet the entry requirements set by the Managed Access Agreement.

Read our historical HPP MAA documents:

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