How are you taking part in this consultation?

You will not be able to change how you comment later.

You must be signed in to answer questions

  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

  • Question on Consultation

    Are the recommendations sound and a suitable basis for guidance to the NHS?

  • Question on Consultation

    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of age, disability, gender reassignment, pregnancy and maternity, race, religion or belief, sex or sexual orientation?
The content on this page is not current guidance and is only for the purposes of the consultation process.

Pegzilarginase for treating arginase-1 deficiency in people 2 years and over

Closed for comments This consultation ended on at   Request commenting lead permission

1 Recommendations

1.1

Pegzilarginase is not recommended, within its marketing authorisation, for treating arginase‑1 deficiency (also called hyperarginaemia) in people 2 years and over.

1.2

This recommendation is not intended to affect treatment with pegzilarginase 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

Usual treatment for arginase‑1 deficiency includes dietary protein restrictions, essential amino acid supplementation and ammonia-lowering drugs. Pegzilarginase is the first treatment that specifically treats arginase‑1 deficiency.

Clinical trial evidence shows that pegzilarginase plus usual treatment reduces levels of arginine in the blood compared with placebo plus usual treatment. Evidence also suggests improvements in mobility and mental processing, but this is uncertain because the studies were small and short. So, it is unclear how large these benefits are or how long these improvements will last.

There are also several uncertainties in the economic model, including:

  • whether the number of people grouped by disease severity is similar to that in NHS clinical practice

  • assumptions on how age varies in NHS clinical practice at the start of each disease severity group

  • how pegzilarginase affects body weight and levels of ammonia in the blood

  • how long people stay on pegzilarginase treatment.

Because of the uncertainties in the clinical evidence and economic model, the most likely cost-effectiveness estimates are substantially above the range that NICE considers an acceptable use of NHS resources for highly specialised technologies. So, pegzilarginase is not recommended.