What Is Joenja, and How Does It Work?
The FDA has approved Joenja (leniolisib) for children ages 4 to 11 years who weigh at least 27 kilograms (about 60 pounds) and have activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS). Joenja was previously approved for adults and children 12 years of age and older. This expanded approval makes it the first targeted medicine available for these younger children with APDS in the United States.
APDS is an ultra-rare genetic immune disease. It causes overactivity in a specific protein pathway in the body's immune cells. This overactivity prevents white blood cells from maturing and working normally. As a result, children with APDS often get frequent lung and sinus infections, swollen lymph nodes, and inflammation. Joenja works by blocking the overactive protein, helping restore normal immune function and reduce swelling.
Why Was Joenja Approved?
The FDA expanded the approval of Joenja based on a clinical study in children ages 4 to 11 years who weigh at least 27 kilograms. The study evaluated the safety and blood levels of Joenja in eight young patients over 12 to 24 weeks of treatment.
The trial showed that Joenja led to shrinking of swollen lymph nodes and improved levels of healthy, young immune cells (naïve B cells). Blood levels of the drug in these young children were similar to those seen in older patients who responded well to treatment. Side effects were mostly mild to moderate, and no drug-related serious safety issues were seen in the children. The common side effects reported in this younger group included belly pain, coughing, and lung infections.
What Do I Need to Know About Joenja?
Joenja is taken as a tablet by mouth two times a day, about 12 hours apart. It can be taken with or without food. Your child's healthcare provider will base the tablet strength on your child's body weight.
Joenja can cause serious allergic reactions, including severe allergic responses like anaphylaxis. Seek emergency medical care right away if your child shows signs of a severe allergic reaction, such as hives, swelling, or trouble breathing. Live vaccines may be less effective while taking Joenja, so tell your healthcare provider about any planned shots. Common side effects in children ages 4 to 11 include stomach pain, coughing, and breathing infections. Low white blood cell counts can also happen, though infections related to this were not reported in trials.
Tell your child's healthcare provider if your child has liver problems, as Joenja is not recommended for people with moderate to severe liver issues. Joenja can harm a fetus and pass into breast milk. Females of childbearing age must have a negative pregnancy test before starting Joenja, use effective birth control during treatment and for one week after the last dose, and avoid breastfeeding during treatment and for one week after the last dose. Tell your healthcare provider about all other prescription drugs, over-the-counter medicines, and supplements your child takes, as Joenja can interact with several other drugs.