NDAORALTABLETPriority Review
Approved
Mar 2023
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
1
Data completeness
7/8 — Comprehensive
Priority Review
JOENJA (leniolisib) by Pharming Group is pi3k-delta by blocking the active binding site of pi3k-delta. Approved for activated phosphoinositide 3-kinase delta syndrome. First approved in 2023.
Drug data last refreshed 20h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
JOENJA (leniolisib) is an oral small-molecule PI3K-delta inhibitor approved in March 2023 for Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS), a rare primary immunodeficiency disorder. It works by selectively blocking PI3K-delta signaling, which is hyperactivated in APDS patients due to gain-of-function or loss-of-function genetic variants, thereby normalizing dysregulated B and T cell proliferation and activation.
$0.002BPart D
Peak5.8 years to LOEEarly-stage product in peak lifecycle phase with minimal Part D penetration; commercial team likely small and specialized in rare disease.
Mechanism of Action
PI3K-delta by blocking the active binding site of PI3K-delta. In cell-free isolated enzyme assays, leniolisib was selective for PI3K-delta over PI3K-alpha (28-fold), PI3K-beta (43-fold), and PI3K-gamma (257-fold), as well as the broader kinome. In cell-based assays, leniolisib reduced pAkt pathway…
Indications (1)
Clinical Trials (1)
Long-term Safety and Efficacy of Leniolisib in PIDs With Immune Dysregulation
Started Apr 2025
12 enrolled
PIDs Linked to PI3K
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.