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FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.

PHARMPHAR
  • The U.S. FDA has approved Joenja (leniolisib) for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) who weigh at least 27 kg.
  • This approval makes Joenja the first FDA-approved treatment for this specific pediatric age group in the U.S.
  • New 40 mg and 50 mg twice-daily dosing regimens are expected to be available to eligible patients in October.
  • Pharming submitted a separate supplemental New Drug Application on July 30 to seek approval for lower doses for children aged 4 to 11 weighing between 13 kg and less than 27 kg.
  • The approval is supported by Phase III study data showing reduced lymphadenopathy and increased naïve B cells over 12 weeks.
  • The safety profile for this group was consistent with prior experiences, with all treatment-emergent adverse events being mild to moderate.
  • Common adverse reactions for pediatric patients aged 4 to less than 12 years weighing 27 kg or greater include abdominal pain, cough, and respiratory tract infection.
  • Approximately 63% of pediatric patients in the study developed an absolute neutrophil count between 500 and 1500 cells/microL, with no cases of neutropenia below 500 cells/microL or associated infections.
  • Joenja is a kinase inhibitor indicated for patients 4 years of age and older weighing 27 kg or greater, with specific warnings regarding pregnancy, breastfeeding, live vaccinations, hepatic impairment, and drug interactions.
  • APDS is a rare primary immunodeficiency caused by genetic variants in PIK3CD or PIK3R1 genes, leading to immune dysregulation, recurrent infections, and potential long-term complications like lung damage.
  • Pharming previously received FDA approval for Joenja in adult and pediatric patients 12 years of age and older in March 2023.