Activated Phosphoniositide 3-kinase Delta Syndrome (APDS in children 4–11) (label expansion)
PHARPharming GroupCatalyst overview
Research summary
This catalyst tracks a PDUFA approval decision for Leniolisib in Activated Phosphoniositide 3-kinase Delta Syndrome (APDS in children 4–11) (label expansion). The expected timing is based on the latest available company guidance and may change.
Pediatric trial. 11/21/23- First patient dosed. 12/11/24- Topline data reported. 3/13/25- Global regulatory filing to begin w/ a US submission in H2 2025. 10/1/25- sNDA accepted, priority review granted. PDUFA date of January 31, 2026. 11/06/25:- Reiterated PDUFA 1/31/26; Q1 2026 launch. 02/01/26:- FDA issued CRL; additional pediatric PK data and Type A meeting planned. 03/12/26:- Type A FDA meeting for pediatric label expansion expected in March 2026. 03/24/26:- Japan MHLW approved Joenja for APDS patients aged 4+. 05/07/26:- CIS 2026 posters to present pediatric APDS 4–11 long-term extension interim outcomes. Resubmitted higher-dose pediatric sNDA; lowest-dose filing summer. 06/04/26:- Resubmitted pediatric sNDA accepted; action date Oct 24, 2026. Separate low-weight sNDA planned Summer 2026. 07/30/26:- PDUFA remains October 24, 2026; lower-dose pediatric sNDA planned in coming days.
Verification
Confirm timing and program details against the company announcement or filing.