leniolisib (Joenja)
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Indications
- treatment of immune dysregulation in PI3K delta syndrome* (APDS), formerly known as PASLI disease, in adults & pediatric patients >= 12 years
* activated phosphoinositide 3-kinase delta syndrome
Dosage
- 70 mg BID
Mechanism of action
- inhibits PI3K delta by blocking the active binding site of PI3K delta
- inhibits signaling pathways leading to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, & dysregulation of B-cells & T cells
- reduces spleen size
More general terms
References
- ↑ Rao VK et al A randomized, placebo-controlled phase 3 trial of the PI3Kdelta inhibitor leniolisib for activated PI3Kdelta syndrome Blood. 2023 Mar 2;141(9):971-983 PMID: https://www.ncbi.nlm.nih.gov/pubmed/36399712 PMCID: PMC10163280 Free PMC article