Joenja (leniolisib) is an oral medication that’s used to treat a rare genetic condition called activated phosphoinositide 3-kinase delta syndrome (APDS), which affects your immune system. It’s for people 12 years of age and older who weigh 99 lbs or more. Joenja (leniolisib) can cause inflammation-related side effects such as sinus pressure and headaches.
Activated phosphoinositide 3-kinase delta syndrome (APDS)
When you have APDS, there’s a protein in your immune system called phosphoinositide 3-kinase (PI3K) delta that doesn’t function correctly. As a result, your immune system becomes weak. This raises your chances of getting sick and catching infections.
Joenja (leniolisib) is a kinase inhibitor known as PI3K delta inhibitor. It works by blocking the PI3K delta protein’s activity. This helps regulate your immune system and protect your body from infection.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
First and only medication used to treat APDS
Can be used in children ages 12 years and older who weigh at least 99 lbs
No reports of severe side effects in clinical studies to date
Taken twice a day
Can’t take while pregnant or breastfeeding
Shouldn’t be used if you have severe liver problems
You can take Joenja (leniolisib) with or without food.
If you vomit within one hour of taking Joenja (leniolisib), you’ll need to take another dose of the medication as soon as possible. Lower your chance of nausea and vomiting by taking Joenja (leniolisib) with food.
If you’ve forgotten to take Joenja (leniolisib) and it’s been more than six hours from the usual time you take your medication, wait and take your next dose at your usual time. Set an alarm as a reminder to take your medication to help yourself not miss a dose.
Store Joenja (leniolisib) at room temperature in its original container. Don’t refrigerate Joenja (leniolisib) because this can damage the medication.
Joenja (leniolisib) might interact with certain medications, including St. John's wort and ketoconazole. These medications could change the amount of Joenja (leniolisib) in your body, possibly either making it less likely to work or raising your chances of having side effects. Tell your healthcare provider about all the medications you take, including over-the-counter medications and supplements, before starting Joenja (leniolisib). They can make sure your medications are safe to take together.
If you’re able to become pregnant, use birth control while taking Joenja (leniolisib) until one week after your last dose. It’s important to avoid becoming pregnant during treatment because of the medication’s risk of harm to an unborn baby.
Joenja (leniolisib) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Risk factors: Pregnant women | Women who can have children
Based on animal studies, it’s possible that Joenja (leniolisib) can cause harm or birth defects to an unborn baby when taken during pregnancy. While Joenja (leniolisib) hasn’t been studied in human pregnancy, it’s important to be aware of this risk. If you're able to get pregnant, you must take a pregnancy test before starting this medication. Make sure to use birth control while you’re taking Joenja (leniolisib) as well as 1 week after your last dose. Talk to your healthcare provider if you think you might be pregnant or are wanting to become pregnant.
Talk to your healthcare provider before getting any vaccines while you’re taking Joenja (leniolisib). Your provider can check to see whether the vaccine is safe for you. Certain vaccines might not work as well to protect you from infection if you get them while you’re taking Joenja (leniolisib). These include live vaccines, such as measles, mumps, and rubella (MMR), varicella (chicken pox), yellow fever, and rotavirus vaccines.
Joenja (leniolisib) is a tablet that you take by mouth. The typical dose is 70 mg by mouth twice a day (about every 12 hours).
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Immune Deficiency Foundation. (n.d.). Activated PI3K delta syndrome (APDS).
Koneti Rao, V., et al. (2023). A randomized, placebo-controlled phase 3 trial of the PI3Kδ inhibitor leniolisib for activated PI3Kδ syndrome. Blood.
National Organization for Rare Disorders. (2023). Activated phosphoinositide 3-kinase delta syndrome (APDS).
Pharming Healthcare Inc. (2023). Joenja- leniolisib tablet, film coated [package insert]. DailyMed.
U.S. Food and Drug Administration. (2023). FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome.
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