NICE Guidance: Leniolisib for PI3K delta syndrome

NICE Highly Specialised Technology Evaluations

Published April 23rd, 2025

Detected March 12th, 2026

Summary

NICE has published highly specialised technologies guidance on the use of leniolisib (Joenja) for treating activated phosphoinositide 3-kinase delta syndrome in individuals aged 12 and over. The guidance includes evidence-based recommendations and information on commercial arrangements for NHS organisations.

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What changed

NICE has issued final guidance (HST33) recommending leniolisib (Joenja) for treating activated phosphoinositide 3-kinase delta syndrome in individuals aged 12 years and older. This guidance provides evidence-based recommendations for healthcare professionals and outlines commercial arrangements, including a patient access scheme, for NHS organisations. It also details the process for reporting adverse events via the Yellow Card Scheme to the Medicines and Healthcare products Regulatory Agency.

Healthcare providers are expected to consider this guidance when making treatment decisions, in consultation with patients. Commissioners and providers have a responsibility to ensure funding is available for leniolisib when clinicians and patients wish to use it. The guidance also emphasizes the need for providers to assess and reduce environmental impact where possible. While the guidance is non-binding, it represents NICE's view based on available evidence.

What to do next

Review NICE guidance HST33 regarding leniolisib for PI3K delta syndrome.
Consult with patients and/or their carers regarding treatment options, considering this guidance.
Report any adverse events related to leniolisib use to the MHRA via the Yellow Card Scheme.

Source document (simplified)

Leniolisib for treating activated phosphoinositide 3-kinase delta syndrome in people 12 years and over

Highly specialised technologies guidance
HST33
Published:

23 April 2025

Download guidance (PDF)

Overview

Evidence-based recommendations on leniolisib (Joenja) for treating activated phosphoinositide 3-kinase delta syndrome in people 12 years and over.

Last reviewed: 23 April 2025

Next review: This guidance will be reviewed if there is new evidence that is likely to change the recommendations.

Commercial arrangement

There is a simple discount patient access scheme for leniolisib. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact infoUK@pharming.com for details.

Guidance development process

How we develop NICE highly specialised technologies guidance

Your responsibility

The recommendations in this guidance represent the view of NICE, arrived at after careful consideration of the evidence available. When exercising their judgement, health professionals are expected to take this guidance fully into account, alongside the individual needs, preferences and values of their patients. The application of the recommendations in this guidance is at the discretion of health professionals and their individual patients and do not override the responsibility of healthcare professionals to make decisions appropriate to the circumstances of the individual patient, in consultation with the patient and/or their carer or guardian.

All problems (adverse events) related to a medicine or medical device used for treatment or in a procedure should be reported to the Medicines and Healthcare products Regulatory Agency using the Yellow Card Scheme.

Commissioners and/or providers have a responsibility to provide the funding required to enable the guidance to be applied when individual health professionals and their patients wish to use it, in accordance with the NHS Constitution. They should do so in light of their duties to have due regard to the need to eliminate unlawful discrimination, to advance equality of opportunity and to reduce health inequalities.

Commissioners and providers have a responsibility to promote an environmentally sustainable health and care system and should assess and reduce the environmental impact of implementing NICE recommendations wherever possible.