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
FDA approves Pharming’s Joenja® as first treatment for children with APDS in the U.S.
Sep 9, 2026Pharming Group to participate in September investor conferences
Aug 13, 2026Pharming announces commercial launch of Joenja (leniolisib) in Japan for adult and pediatric patients with APDS aged 4 and older
Aug 4, 2026Pharming Group to participate in Canaccord Genuity 46th Annual Growth Conference
Jul 30, 2026Pharming reports second quarter and first half 2026 financial results and revises year-end guidance; strong Joenja momentum and near-term clinical readouts to support broader therapeutic use