Ultra Orphan Assessment
The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for the above product using the ultra-orphan framework.
Indication under review: Treatment of activated phosphoinositide 3-kinase delta (PI3K-delta) syndrome (APDS) in adult and paediatric patients 12 years of age and older.
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Scottish Government Medicines Policy Branch will notify Health Boards when this medicine is available for prescribing within the ultra-orphan pathway. Meantime any requests to access treatment should be considered through local non-formulary processes.
Medicine details
- Medicine name:
- leniolisib (Joenja)
- SMC ID:
- SMC2836
- Indication:
Treatment of activated phosphoinositide 3-kinase delta (P13Kδ) syndrome (APDS) in adult and paediatric patients 12 years of age and older.
- Pharmaceutical company
- Pharming Group NV
- BNF chapter
- Nutrition and blood
- Submission type
- Ultra-orphan initial assessment
- Date Published
- 08 December 2025