JOENJA (leniolisib) 40 mg and 50 mg tablets
Activated phosphoinositide 3-kinase delta syndrome · Children aged 4 to 11 years weighing 27 kg or more
PHAR · Pharming Group
Regulatory designations
Full timeline
6 milestonesFDA approved JOENJA pediatric label expansion
FDA approved the resubmitted pediatric sNDA for JOENJA 40 mg and 50 mg twice-daily dosing in children aged 4 to 11 years who weigh at least 27 kg and have activated phosphoinositide 3-kinase delta syndrome. The approval makes JOENJA the first FDA-approved treatment for APDS in this age group.
Pharming — FDA approval of JOENJA for children with APDS↗FDA accepted the sNDA resubmission
The resubmission seeks approval of 40 mg and 50 mg twice-daily dosing for children aged 4 to 11 years weighing at least 27 kg and includes additional production-batch analytical data requested by FDA.
Pharming — Resubmission acceptance↗Pharming held a Type A meeting with FDA
The meeting followed the first-cycle CRL and informed the scope of the pediatric sNDA resubmission.
Pharming — Resubmission acceptance and regulatory history↗FDA issued a Complete Response Letter in the first review cycle
FDA requested additional pediatric pharmacokinetic information for lower-weight groups and additional data and clarification regarding a production-batch testing method.
Pharming — Complete Response Letter↗Related News
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Sep 25, 2026Pharming announces U.S. FDA acceptance and Priority Review of sNDA for lower doses of Joenja® to treat children with APDS
Sep 22, 2026Pharming announces positive Phase II topline data for leniolisib in PIDs with immune dysregulation accepted as late-breaking abstract at ESID 2026
Sep 11, 2026FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.
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