Leniolisib
Treatment of symptomatic activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adults and adolescents aged 12 years and older.
Decision on record
- Meeting Jul 2025 Recommended Symptomatic activated PI3K delta syndrome in adults and adolescents aged 12+
Access path
- Jul 2025Recommended · restricted
vs standard of care (placebo/best supportive care)
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the Section 100 (Highly Specialised Drugs Program) listing of leniolisib for the treatment of symptomatic APDS in adults and adolescents aged 12 years and older. In making this recommendation, the PBAC recognised the high unmet clinical need for treatment options for patients with APDS noting the condition results in substantial impacts on quality of life for patients and reduced life-expectancy.PSD · Jul 2025
OFFICIAL Public Summary Document - July 2025 PBAC meeting lymphadenopathy and risk of lymphoma. The PBAC considered that the incremental cost-effectiveness was uncertain due to the limited data informing the economic model, but that in the context of this rare and life-limiting disease, leniolisib would be considered acceptably cost-effective with a price reduction that would result in an acceptable cost per patient per year.PSD · Jul 2025
6.48 The submission presented a cost-utility analysis (CUA) that compared leniolisib + SoC with SoC alone based on one direct randomised trial (Study 2201 Part II), with additional clinical data from Study 2201E, the leniolisib early access program (EAP) and the ESID-APDS registry.PSD · Jul 2025
6.49 The key components of the economic model are presented in Table 14.PSD · Jul 2025
6.43 The submission described leniolisib as superior in terms of effectiveness compared with SoC and non-inferior in terms of safety compared to SoC.PSD · Jul 2025
6.44 The Sub-Committees agreed with the Commentary that the therapeutic conclusion presented in the submission was partially supported by the evidence presented in the submission because:PSD · Jul 2025
6.3 The PBAC noted and welcomed the input from individuals (1), health care professionals (2) and organisations (2) via the Consumer Comments facility on the PBS website. The comments described how debilitating and isolating APDS is given the frequency of infections resulting in limited participation at work and school.PSD · Jul 2025
The input described the benefits of treatment with leniolisib including reduced frequency of illness which has resulted in avoiding stem cell or lung transplants and allowed the patient to leave the house. The patient described the increase in quality of life in terms of physical, emotional and mental health. The patient described the side effects of leniolisib, although hard to distinguish from infection, are manageable.PSD · Jul 2025
Cost-effectiveness
ICER values are redacted (commercial-in-confidence); only that a cost-effectiveness analysis versus standard of care was performed is stated.
Decision context
PopulationAdults 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.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2025 | Recommended · restricted | standard of care (placebo/best supportive care) | — | RCT · Other |
Consumer voice
Consumer input from one patient, two healthcare professionals, and two organisations (ASCIA and AusPIPs Inc) described APDS as debilitating and isolating, with leniolisib providing benefits including reduced infection frequency, improved quality of life, and avoiding need for transplants, though access barriers and continued need for supportive therapies were noted.
The comments described how debilitating and isolating APDS is given the frequency of infections resulting in limited participation at work and school. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC requires confirmed pathogenic variants in PIK3CD/PIK3R1 and demonstrated APDS symptoms; TGA label does not specify genetic confirmation requirement.