FDA Approval

FDA Expands Joenja Approval to Children Aged 4 to 11 Years With APDS

Edited by:
Anthony Calabro, MA

Key Highlights

  • The expanded indication covers children aged 4 to 11 years weighing at least 27 kg.
  • Pediatric dosing is weight based at 40, 50, or 70 mg twice daily.
  • Pharmacokinetic exposure did not differ clinically between patients younger and older than 12 years.
  • Common adverse effects included abdominal pain, respiratory tract infection, and diarrhea.

On September 11, the FDA approved leniolisib (Joenja; Pharming Healthcare, Inc) tablets for activated phosphoinositide 3-kinase delta syndrome (APDS) in children aged 4 to 11 years who weigh at least 27 kg. Leniolisib, first approved in 2023 for patients aged 12 years or older, is the first approved APDS treatment for this younger population.

Safety and pharmacokinetic evidence for the expanded indication came from LE 3301 (NCT05438407), a single-arm, open-label study in which 8 children received weight-based leniolisib. Pharmacokinetic findings showed no clinically significant difference between patients younger and older than 12 years.

The original approval was supported by Study 2201 (NCT02435173), a 12-week, blinded, randomized, placebo-controlled trial involving 31 patients aged 12 years or older. By day 85, leniolisib recipients experienced reduced lymph node size and a 37% improvement in naïve B-cell counts compared with placebo.

The most common adverse effects among children aged 4 to 11 years were abdominal pain, respiratory tract infection, diarrhea, headache, cough, nausea, rhinitis, and alopecia. Patients with moderate to severe hepatic impairment should not receive leniolisib.

Children aged 4 to 11 years should receive a weight-based dose of 40, 50, or 70 mg orally twice daily, approximately 12 hours apart. The release did not provide the weight thresholds corresponding to each dose or specify a treatment duration. For patients aged 12 years or older, the approved dosage is 70 mg orally twice daily.

Reference
US Food and Drug Administration. FDA approves first treatment for children aged 4-11 years with APDS, a rare genetic disorder of the immune system. Accessed September 15, 2026. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-children-aged-4-11-years-apds-rare-genetic-disorder-immune-system