Curie Brief
Turn on cookies to sign in
Signing in saves your progress to your Curie account. We can only do that with cookies on — turn them on to continue.
News
The FDA has approved Joenja (leniolisib) for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare genetic immune disorder — making it the only approved treatment for this age group. The approval covers children weighing at least 27 kg, with dosing tailored by weight. Pharming is already pursuing approval for even younger, lighter patients.
The FDA has approved Joenja (leniolisib) for children aged 4 to 11 years diagnosed with activated phosphoinositide 3-kinase delta syndrome (APDS) — a rare primary immunodeficiency caused by genetic variants in PIK3CD or PIK3R1. This makes Joenja the only FDA-approved treatment specifically for this younger pediatric age group, building on its existing approval for patients 12 and older since March 2023.
The approval is based on a phase 3 study of eight children, which showed reductions in lymphadenopathy and increases in naïve B cells over 12 weeks — signs that the underlying immune defect was being addressed. Dosing (40 mg, 50 mg, or 70 mg, taken twice daily) is determined by the child's weight, and the drug is approved for those weighing at least 27 kg. Pharming has already submitted a new application to extend approval to children weighing as little as 13 kg.
Key Takeaways:
Why it matters: APDS can disrupt schooling, social life, and daily activities in young children, and without treatment, it can lead to irreversible complications. This approval gives clinicians a disease-modifying option earlier in the disease course — a significant step forward for a condition that previously had no approved therapy for this age group.
