Joenja (leniolisib)
An approved treatment for Activated PI3K Delta Syndrome.
The same compound appears under different names depending on the context. Here is how to identify Leniolisib wherever you encounter it, plus the key facts at a glance.
- Generic name
- Leniolisib
- Brand name
- Joenja
- Development code
- CDZ173
- Drug class
- PI3K-delta inhibitor
- Manufacturer
- Pharming Group
- How it's taken
- A tablet taken twice a day, about 12 hours apart, with or without food.
A twice-daily pill and the first treatment approved for activated PI3K delta syndrome (APDS): approved March 24, 2023 for people 12 and older, and on September 11, 2026 for children 4 to 11 who weigh at least 27 kg.
The decision is expected by January 30, 2027. One email when it is public, with what it means in plain language, plus new trials for Activated PI3K Delta Syndrome and FDA news. Unsubscribe anytime.
You'll get new recruiting trials, changes to trials, FDA decisions and condition news for Activated PI3K Delta Syndrome. Turn any of these off from the link in every email.
Where Leniolisib fits
The only approved treatment aimed at the cause of APDS, often used alongside immunoglobulin replacement and preventive antibiotics.
How Leniolisib works
In APDS a change in PIK3CD or PIK3R1 leaves the PI3K-delta enzyme switched on too much, so B cells stay immature, T cells wear out and lymph tissue swells. Leniolisib blocks PI3K-delta, turning that signal back down. In its trial, swollen lymph nodes shrank and naive B cells, a sign of a healthier immune system, rose.
Mechanism: Oral inhibitor of PI3K-delta, the immune cell enzyme that is overactive in activated PI3K delta syndrome
Side effects and safety
- Harm to an unborn baby. Pregnancy is checked before starting, and effective birth control is used during treatment and for 1 week after the last dose.
- Live vaccines. Live, weakened vaccines may work less well if given during treatment.
- Allergic reactions. Serious allergic reactions, including anaphylaxis, have been reported since approval. Stop and get care if they occur.
- Pregnancy check before starting
- Review of other medicines for interactions, especially strong CYP3A4 inhibitors and inducers
- Liver function, since it is not recommended with moderate to severe liver impairment
Joenja has no boxed warning. It may harm an unborn baby, so a pregnancy check is done before starting and effective birth control is used until 1 week after the last dose. Live vaccines may work less well during treatment, and serious allergic reactions including anaphylaxis have been reported since approval. It is not recommended with moderate to severe liver impairment, and several drug combinations are avoided. In the main trial, headache (24% against 20% on placebo), sinusitis (19% against 0%) and eczema (14% against 0%) were the most common side effects; in children aged 4 to 11, belly pain, cough and respiratory infection were most common.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Leniolisib
A tablet taken twice a day, about 12 hours apart, with or without food. People 12 and older take 70 mg. Children 4 to 11 take 40 mg if they weigh 27 to under 38 kg, 50 mg if 38 to under 45 kg, and 70 mg if 45 kg or more.
Availability and cost
Only available as the brand-name product.
Help paying for Joenja
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Insurance and case manager help
A Care Coordinator helps you understand coverage, access and financial resources for Joenja. An APDS Clinical Educator gives one-on-one support, and specialty pharmacists help with prior authorization and refills. You need to sign a patient consent form to get these services.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source - Bridge or quick-start supply
The Joenja Starter program gives eligible patients quick access to Joenja while the doctor's office works with the insurance company to set up coverage. Eligibility rules are not published.
The official page does not say who qualifies. Ask the program. · source - Free medicine program
The enrollment form has a Product Assistance Program section for patients with a documented APDS diagnosis. It covers up to a 30-day supply with no refills. Pharming says other terms apply but does not publish them.
The official page does not say who qualifies. Ask the program. · source
Good to know: The enrollment form mentions copay assistance for eligible patients, but no official page states the copay terms, so it is not listed here. Prescriptions are filled through PANTHERx Rare Pharmacy.
Clinical trial results
In a 12-week randomized, placebo-controlled trial (NCT02435173) of 31 people 12 and older (21 on Joenja, 10 on placebo), the size of the largest lymph nodes shrank more on Joenja (a log-scale change of 0.27 against 0.02), and naive B cells rose 37.4 percentage points against 0.1. The pediatric approval rests on a study of 8 children aged 4 to 11 (NCT05438407) that looked at safety and drug levels.
Development history
Developed by Novartis as CDZ173 and licensed to Pharming. The FDA approved Joenja on March 24, 2023 for people 12 and older who weigh at least 45 kg, the first treatment for APDS. After a complete response letter on January 30, 2026, a resubmission led to approval on September 11, 2026 for children 4 to 11 weighing at least 27 kg, with new 40 mg and 50 mg tablets. The FDA is reviewing lower doses for children 4 to 11 who weigh 13 to 27 kg, with a decision due January 30, 2027.
Explore Activated PI3K Delta Syndrome trials
Common questions about Leniolisib
▸What is Leniolisib (Joenja)?
A twice-daily pill and the first treatment approved for activated PI3K delta syndrome (APDS): approved March 24, 2023 for people 12 and older, and on September 11, 2026 for children 4 to 11 who weigh at least 27 kg.
▸How does Leniolisib work?
In APDS a change in PIK3CD or PIK3R1 leaves the PI3K-delta enzyme switched on too much, so B cells stay immature, T cells wear out and lymph tissue swells. Leniolisib blocks PI3K-delta, turning that signal back down. In its trial, swollen lymph nodes shrank and naive B cells, a sign of a healthier immune system, rose.
▸What are the side effects of Leniolisib?
Joenja has no boxed warning. It may harm an unborn baby, so a pregnancy check is done before starting and effective birth control is used until 1 week after the last dose. Live vaccines may work less well during treatment, and serious allergic reactions including anaphylaxis have been reported since approval. It is not recommended with moderate to severe liver impairment, and several drug combinations are avoided. In the main trial, headache (24% against 20% on placebo), sinusitis (19% against 0%) and eczema (14% against 0%) were the most common side effects; in children aged 4 to 11, belly pain, cough and respiratory infection were most common.
▸How is Leniolisib taken?
A tablet taken twice a day, about 12 hours apart, with or without food. People 12 and older take 70 mg. Children 4 to 11 take 40 mg if they weigh 27 to under 38 kg, 50 mg if 38 to under 45 kg, and 70 mg if 45 kg or more.
▸Is Leniolisib FDA approved?
Yes, Leniolisib (Joenja) is FDA approved (2023) for the treatment of Activated PI3K Delta Syndrome.
▸Can children take Joenja?
Yes, from age 4 if they weigh at least 27 kg, since the FDA's September 11, 2026 approval. Children 4 to 11 take 40 mg, 50 mg or 70 mg twice a day depending on weight. Lower doses for children weighing 13 to 27 kg are under FDA review, with a decision due January 30, 2027.
▸Does Joenja replace immunoglobulin therapy?
Not automatically. Joenja targets the overactive enzyme behind APDS, and in its trial lymph nodes shrank and naive B cells rose, but decisions about immunoglobulin replacement and antibiotics are made with the immunologist based on each person's infections and lab results.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2026-09. JOENJA (leniolisib) tablets: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=eefbcef8-2130-4654-8f29-b58077e08212
- U.S. Food and Drug Administration · 2026-09-11. FDA approves first treatment for children aged 4 to 11 years with APDS. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-children-aged-4-11-years-apds-rare-genetic-disorder-immune-system
- Pharming Group · 2023-03-24. Pharming announces FDA approval of Joenja for APDS. https://www.pharming.com/files/leniolisib-pdufa-approval-en-24mar23.pdf