IN THE RANDOMIZED, PLACEBO-CONTROLLED PIVOTAL TRIAL
Joenja safety profile1,2
Not actual patients.
The safety of Joenja is based on data from both the placebo-controlled pivotal trial and the interim results from the OLE study
Adverse reactions reported by ≥2 Joenja-treated patients and more frequently than placebo
- No serious adverse drug reactions
were reported - No patients withdrew due to an adverse
drug reaction - The most common adverse reactions (>10%) were headache, sinusitis, and dermatitis atopic
*Dermatitis atopic: including dermatitis atopic and eczema.
†Tachycardia: including tachycardia and sinus tachycardia.
ANC, absolute neutrophil count; OLE, open-label extension.
Seven (33%) patients receiving JOENJA developed an ANC between 500 and 1500 cells/microL. No patients developed an ANC <500 cells/microL and there were no reports of infection associated with neutropenia.
About APDS
APDS is a rare, primary immunodeficiency.
Hyperactivity along the Pl3Kδ signaling
pathway disrupts immune cell balance,
causing immune deficiency and
immune dysregulation.1,2,4,7
Hyperactivity along the Pl3Kδ signaling
pathway disrupts immune cell balance,
causing immune deficiency and
immune dysregulation.1,2,4,7
The extension
study
The long-term safety and tolerability
of Joenja is under investigation in
the open-label extension study with
some patients receiving treatment
for as long as 5 years.1,6
of Joenja is under investigation in
the open-label extension study with
some patients receiving treatment
for as long as 5 years.1,6
References: 1. Joenja (leniolisib). Prescribing information. Pharming Healthcare, Inc; 2023. 2. 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 3. Data on file. Pharming Healthcare, Inc. 4. 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 5. 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 6. US National Library of Medicine. ClinicalTrials.gov/NCT02859727. 7. 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
Registry. Front Immunol. 2018;9:543. doi:10.3389/fimmu.2018.00543 5. 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 6. US National Library of Medicine. ClinicalTrials.gov/NCT02859727. 7. 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
