APDS

3
Pipeline Programs
1
Companies
2
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
2
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
2100%

On Market (1)

Approved therapies currently available

Pharming Group
JOENJAApproved
leniolisib
Pharming Group
oral2023

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Pharming Group
Pharming GroupLEIDEN, Netherlands
2 programs
2
1
LeniolisibPhase 3Small Molecule1 trial
LeniolisibPhase 3Small Molecule1 trial
Active Trials
NCT05693129Active Not RecruitingEst. Oct 2026
NCT05438407Active Not RecruitingEst. Dec 2025

Trial Timeline

Clinical trial activity over time

2023
2024
2025
2026
Pharming GroupLeniolisib
Pharming GroupLeniolisib

Clinical Trials (2)

Pediatric Patients Aged 1 to 6 Years With APDS

Start: Aug 2023Est. completion: Oct 2026
Phase 3Active Not Recruiting

Pediatric Patients Aged 4 to 11 Years With APDS

Start: Feb 2023Est. completion: Dec 2025
Phase 3Active Not Recruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space