Leniolisib
- Generic Name
- Leniolisib
- Brand Names
- Joenja
- Drug Type
- Small Molecule
- Chemical Formula
- C21H25F3N6O2
- 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...
- 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
- Associated Therapies
- -
Leniolisib for Immune Dysregulation in PIDs
- 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
- 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
- First Posted Date
- 2023-01-20
- Last Posted Date
- 2024-12-17
- Lead Sponsor
- Pharming Technologies B.V.
- Target Recruit Count
- 15
- 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
Pediatric Patients Aged 4 to 11 Years With APDS
- First Posted Date
- 2022-06-30
- Last Posted Date
- 2024-07-23
- Lead Sponsor
- Pharming Technologies B.V.
- Target Recruit Count
- 15
- Registration Number
- NCT05438407
- Locations
- 🇺🇸
Children's Healthcare of Atlanta, Atlanta, Georgia, United States
🇺🇸University of California Los Angeles, Los Angeles, California, United States
🇫🇷Necker Hospital Paris, Paris, France
Drug Development Updates
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