Drug updated on 4/16/2024
| Dosage Form | Tablet (oral; 70 mg) |
| Drug Class | Kinase inhibitors |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.
Summary
- Leniolisib (Joenja) is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome in adult and pediatric patients aged 12 years and older. The medication presents a favorable safety profile, with fewer study treatment-related adverse events reported compared to placebo.
- A study from a randomized controlled trial was reviewed to gather information about Joenja's safety and effectiveness.
- In terms of effectiveness, leniolisib significantly outperformed the placebo in all reported outcomes including reduction in lymph node size, increase in the percentage of naive B cells, and decrease in spleen volume. These results underscore its proficiency in addressing key pathological features associated with APDS such as lymphadenopathy, immune dysregulation, and deficiency.
- The comparison was made only against a placebo; no direct comparison was made between leniolisib (Joenja) and other medications used for treating APDS. However, it demonstrated significant improvement over the inactive treatment option.
- The population targeted by the study were patients with ADPS who are at least 12 years old, which provides valuable insights into Joenja's applicability across both adult and pediatric age groups suffering from ADPS.
- No subgroup analyses based on age or other demographic or clinical characteristics were mentioned, limiting understanding of differential impacts, if any, among these subgroups while using leniolisib (Joenja).
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Joenja (leniolisib) Prescribing Information. | 2023 | Pharming Group N.V., Leiden, The Netherlands |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome. | 37Subjects F: 49% M: 51% | 2023 | Blood |
Document Title
Sex Distribution:
F:49%
M:51%
37Subjects
Year:
2023
Source:Blood
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Clinical practice guideline for activated phosphatidyl inositol 3-kinase-delta syndrome in Japan. | 2023 | Immunological Medicine |