PBAC's latest decision on Leniolisib: Recommended with restriction (2025). Considered for Treatment of symptomatic activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adults and adolescents aged 12 years and older.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2025
Submissions
1
2025 → 2025
Eligible population
Adults and adolescents aged 12 years and older with symptomatic APDS confirmed by genomic or genetic testing (pathogenic or likely pathogenic variant in PIK3CD or PIK3R1 gene) and demonstrated APDS-associated symptoms or current therapeutic intervention for APDS complications.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Other
Key trials
Study 2201
Comparator
standard of care (placebo/best supportive care)
Economic model
CUA
ICER note
ICER values are redacted (commercial-in-confidence); only that a cost-effectiveness analysis versus standard of care was performed is stated.