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Pharming announces U.S. FDA acceptance of sNDA resubmission for Joenja® (leniolisib) to treat children aged 4 to 11 years with APDS

PHAR
  • The U.S. FDA accepted Pharming Group's resubmitted supplemental New Drug Application (sNDA) for Joenja (leniolisib) to treat children aged 4 to 11 with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS).
  • If approved, Joenja will become the first U.S. treatment for this rare primary immunodeficiency in the specified pediatric age group.
  • The resubmission seeks approval for 40 mg \& 50 mg twice-daily dosing for pediatric patients weighing 27 kg or more.
  • A separate sNDA targeting lower doses for pediatric patients weighing less than 27 kg is planned for the second half of 2026.
  • The FDA assigned a Prescription Drug User Fee Act (PDUFA) target action date of October 24, 2026.
  • The application includes additional data on analytical methods for production batch testing following a Complete Response Letter received in January 2026.
  • Phase III study data showed improvements in lymphadenopathy \& naïve B cell counts over 12 weeks, with all treatment-emergent adverse events reported as mild to moderate.
  • Joenja previously received FDA approval in March 2023 for APDS treatment in patients aged 12 years \& older.
  • APDS is a genetic disorder caused by variants in PIK3CD or PIK3R1 genes, leading to immune cell dysfunction \& symptoms like recurrent infections \& lymphoproliferation.
  • Leniolisib is currently under regulatory review for APDS in Canada \& other countries, with additional Phase II trials evaluating its use in other primary immunodeficiencies.