We are pleased to share an important update from National Institute for Health and Care Excellence (NICE) regarding a potential new treatment option for people aged two years and older living with arginase 1 deficiency (ARG1d) in England.

NICE have reviewed all the evidence submitted relating to pegzilarginase. The committee has now reached the decision to recommend pegzilarginase for use within the NHS, subject to final guidance being confirmed.

What this could mean for our community

If the recommendation is confirmed, pegzilarginase will become the first disease modifying treatment option for people aged two years and older living with ARG1d in England. This represents a potentially significant step forward for families affected by this rare condition.

Important note

A final consultation period is now ongoing, during which appeals can be lodged. It is expected that a final, definite decision will be published early March. We will continue to monitor developments closely and share updates as soon as more information becomes available.

If you have questions or would like support, please contact our team or speak with your clinical specialist.