Leniolisib

Generic Name: Leniolisib

Brand Names: Joenja

Drug Type: Small Molecule

Chemical Formula: C_₂₁H_₂₅F_₃N_₆O_₂

CAS Number: 1354690-24-6

Unique Ingredient Identifier: L22772Z9CP

Background: Leniolisib is a potent and selective inhibitor of phosphoinositide 3-kinase δ (PI3Kδ). The FDA approved leniolisib on March 24, 2023, making it the first treatment for activated phosphoinositide 3-kinase delta syndrome (APDS). APDS is a primary immunodeficiency caused by mutations in genes encoding the PI3Kδ, thereby increasing the activity of PI3Kδ, causing immune dysfunction, and elevating susceptibility to infections. Leniolisib works to inhibit hyperactive PI3Kδ. Investigations for using leniolisib in primary Sjögren’s syndrome are ongoing.

Indication: Leniolisib is indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

Associated Conditions: Activated PI3 kinase delta syndrome

Clinical Trials

Leniolisib for Immune Dysregulation in CVID

Phase 2

Recruiting

Conditions: Common Variable Immunodeficiency (CVID)

Interventions: Drug: Leniolisib

First Posted Date: 2025-03-26

Last Posted Date: 2025-04-08

Lead Sponsor: Pharming Technologies B.V.

Target Recruit Count: 20

Registration Number: NCT06897358

Locations: 🇺🇸
Lahey Hospital & Medical Center, Burlington, Massachusetts, United States

Leniolisib for Immune Dysregulation in PIDs

Phase 2

Recruiting

Conditions: Primary Immunodeficiency Disorders (PIDs)

Interventions: Drug: Leniolisib

First Posted Date: 2024-08-12

Last Posted Date: 2024-11-12

Lead Sponsor: Pharming Technologies B.V.

Target Recruit Count: 12

Registration Number: NCT06549114

Locations: 🇺🇸
National Institute of Health, Bethesda, Maryland, United States

An Open-Label Study to Assess the Safety & Efficacy of Leniolisib in Japanese Patients With APDS

Phase 3

Recruiting

Conditions: APDS Gene Mutation

Interventions: Drug: Leniolisib

First Posted Date: 2024-02-08

Last Posted Date: 2024-02-08

Lead Sponsor: Pharming Technologies B.V.

Target Recruit Count: 3

Registration Number: NCT06249997

Locations: 🇯🇵
Hiroshima University Hospital, Hiroshima, Hiroshima City, Japan
🇯🇵
Tokyo Medical And Dental University Hospital, Tokyo, Bunkyo-ku, Japan

Pediatric Patients Aged 1 to 6 Years With APDS

Phase 3

Active, not recruiting

Conditions: APDS

Interventions: Drug: Leniolisib

First Posted Date: 2023-01-20

Last Posted Date: 2025-04-23

Lead Sponsor: Pharming Technologies B.V.

Target Recruit Count: 16

Registration Number: NCT05693129

Locations: 🇺🇸
University of California Los Angeles, Los Angeles, California, United States
🇺🇸
National Institutes of Health, Bethesda, Maryland, United States
🇺🇸
The University of North Carolina, Chapel Hill, North Carolina, United States

and more 7 locations

Pediatric Patients Aged 4 to 11 Years with APDS

Phase 3

Active, not recruiting

Conditions: APDS

Interventions: Drug: Leniolisib

First Posted Date: 2022-06-30

Last Posted Date: 2025-03-12

Lead Sponsor: Pharming Technologies B.V.

Target Recruit Count: 15

Registration Number: NCT05438407

Locations: 🇯🇵
Institute of Science Tokyo Hospital, Tokyo, Japan
🇯🇵
Kyoto University Hospital, Kyoto, Japan
🇺🇸
University of California Los Angeles, Los Angeles, California, United States

and more 4 locations