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Joenja

The first and only therapy that is designed to correct the underlying immune defect in APDS1-4

APDS, activated PI3Kδ syndrome.

About Joenja

Joenja is the first and only FDA-approved
therapy indicated for the treatment
of APDS. Joenja helps address both immune
deficiency AND immune dysregulation.5

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Clinical Data

Joenja demonstrated significant efficacy
and safety tolerability in a Phase 3,
randomized controlled clinical trial.1

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Access & Patient Support

Obtain information regarding access to
Joenja, the APDS Assist program, and other
helpful resources for your patients with
APDS.

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About APDS

APDS is a rare, primary immunodeficiency.
Hyperactivity along the PI3Kδ signaling
pathway disrupts immune cell balance,
causing immune deficiency and
immune dysregulation.1,2,5,6

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Valuable Updates

Your resource for clinical developments and important information regarding APDS and Joenja.

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Not actual patients.

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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. 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 6. 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

Indications and Usage

JOENJA® (leniolisib) is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 4 years of age and older and weighing 27 kg or more.

Indications and Usage

JOENJA® (leniolisib) is a kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 4 years of age and older and weighing 27 kg or more.

Important Safety Information

Verify pregnancy status in females of reproductive potential prior to initiating treatment with JOENJA.

JOENJA may cause fetal harm when administered to a pregnant woman. Advise patients of the potential risk to a fetus and to use highly effective methods of contraception during treatment with JOENJA and for 1 week after the last dose. Additionally, advise women not to breastfeed during treatment with JOENJA and for 1 week after the last dose.

Live, attenuated vaccinations may be less effective if administered during JOENJA treatment.

JOENJA may cause hypersensitivity reaction(s), including anaphylaxis. Advise patients to discontinue JOENJA and to seek immediate medical attention if they develop any signs and symptoms of serious allergic reactions.

Use of JOENJA in patients with moderate to severe hepatic impairment is not recommended. There is no recommended dosage for patients weighing less than 27 kg.

Avoid co-administration of JOENJA with other medications known to be strong CYP3A4 inhibitors, strong or moderate CYP3A4 inducers, or BCRP, OATP1B1, and OATP1B3 substrates.

Adult and pediatric patients 12 years of age and older: The most common adverse reactions (incidence >10%) seen in clinical trials were headache, sinusitis, atopic dermatitis, and weight gain.

Pediatric patients 4 to 11 years of age: The most common adverse reactions (incidence > 10%) were headache, cough, pyrexia, vomiting, diarrhea, and sinusitis.

Seven (33%) patients in the adult and pediatric patients 12 years and older (Study 2201) and 10 (47.6%) of pediatric patients 4 to 11 years of age (Study 3301 Part 1) receiving JOENJA developed an absolute neutrophil count (ANC) between 500 and 1500 cells/microL. No patients in either study developed an ANC <500 cells/microL and there were no reports of infection associated with neutropenia.

Before prescribing JOENJA, please read the full Prescribing Information.


Joenja is available in 40 mg, 50 mg, and 70 mg tablets.