Joenja is the first and only selective PI3Kδ inhibitor indicated for the treatment of APDS1-4
Joenja is DESIGNED TO CORRECT the underlying immune defect caused by APDS to help normalize the hyperactive PI3Kδ Pathway4-6
AKT/PKB, protein kinase B; FOXO, forkhead box O; mTOR, mammalian target of rapamycin; p85α, the regulatory subunit of the PI3Kδ enzyme; p110δ, the catalytic subunit of the PI3Kδ enzyme; PDK1, phosphoinositide-dependent kinase 1; PIP2, phosphatidylinositol (4,5)-bisphosphate; PIP3, phosphatidylinositol (3,4,5)-trisphosphate.
Note: Illustration does not include all steps in the signaling pathway.
Joenja is designed to correct the underlying immune
defect caused by APDS to help normalize the
hyperactive PI3Kδ pathway4-6
defect caused by APDS to help normalize the
hyperactive PI3Kδ pathway4-6
Joenja facilitates a balanced PI3Kδ pathway to support proper immune function1,7
Joenja mechanism of action
This video provides a detailed illustration of the mechanism of action for
Joenja and shows how Joenja addresses both immune deficiency AND
immune dysregulation to help normalize immune balance.
Joenja and shows how Joenja addresses both immune deficiency AND
immune dysregulation to help normalize immune balance.
About APDS
APDS is a rare, primary immunodeficiency. Hyperactivity along the Pl3Kδ signaling
pathway disrupts immune cell balance,
causing immune deficiency and
immune dysregulation.1,2,7,8
pathway disrupts immune cell balance,
causing immune deficiency and
immune dysregulation.1,2,7,8
Clinical Data
Joenja demonstrated significant
efficacy and safety tolerability in a
Phase 3, randomized controlled
clinical trial.1
efficacy and safety tolerability in a
Phase 3, randomized controlled
clinical trial.1
References: 1. Joenja (leniolisib). Prescribing information. Pharming Healthcare, Inc; 2023. 2. Maccari ME, Abolhassani H, Aghamohammadi A, et al. Disease evolution and response to rapamycin in activated phosphoinositide 3-kinase δ syndrome: The European Society for Immunodeficiencies-Activated Phosphoinositide 3-Kinase δ Syndrome Registry. Front Immunol. 2018;9:543. doi:10.3389/
fimmu.2018.00543 3. Rao VK, Webster S, Dalm VASH, et al. Effective “activated PI3Kδ syndrome”—targeted therapy with the PI3Kδ inhibitor leniolisib. Blood. 2017;130(21):2307-2316. doi:10.1182/
blood-2017-08-801191 4. Data on file. Pharming Healthcare, Inc. 5. Fruman DA, Chiu H, Hopkins BD, Bagrodia S, Cantley LC, Abraham RT. The PI3K pathway in human disease. Cell. 2017;170(4):605-635. doi:10.1016/j.cell.2017.07.029 6. Okkenhaug K, Vanhaesebroeck B. PI3K in lymphocyte development, differentiation and activation. Nat Rev Immunol. 2003;3(4):317-330. doi:10.1038/nri1056 7. Rao VK, Webster S, Šedivá A, et al. A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome. Blood. 2023;141(9):971-983. doi:10.1182/blood.2022018546 8. Angulo I, Vadas O, Garçon F, et al. Phosphoinositide 3-kinase δ gene mutation predisposes to respiratory infection and airway damage. Science. 2013;342(6160):866-871. doi:10.1126/science.1243292
fimmu.2018.00543 3. Rao VK, Webster S, Dalm VASH, et al. Effective “activated PI3Kδ syndrome”—targeted therapy with the PI3Kδ inhibitor leniolisib. Blood. 2017;130(21):2307-2316. doi:10.1182/
blood-2017-08-801191 4. Data on file. Pharming Healthcare, Inc. 5. Fruman DA, Chiu H, Hopkins BD, Bagrodia S, Cantley LC, Abraham RT. The PI3K pathway in human disease. Cell. 2017;170(4):605-635. doi:10.1016/j.cell.2017.07.029 6. Okkenhaug K, Vanhaesebroeck B. PI3K in lymphocyte development, differentiation and activation. Nat Rev Immunol. 2003;3(4):317-330. doi:10.1038/nri1056 7. Rao VK, Webster S, Šedivá A, et al. A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome. Blood. 2023;141(9):971-983. doi:10.1182/blood.2022018546 8. Angulo I, Vadas O, Garçon F, et al. Phosphoinositide 3-kinase δ gene mutation predisposes to respiratory infection and airway damage. Science. 2013;342(6160):866-871. doi:10.1126/science.1243292
