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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 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
Related News

Pharming Group Initiates Phase II Trial of Leniolisib for CVID with Immune Dysregulation

• Pharming Group has dosed the first patient in a Phase II clinical trial evaluating leniolisib for common variable immunodeficiency (CVID) with immune dysregulation, representing a significant expansion beyond its approved APDS indication. • The multi-center trial will enroll approximately 20 patients aged 12 and older across sites in the US, UK, and EU, targeting a condition with an 11-fold higher mortality rate compared to CVID patients with infectious manifestations alone. • CVID with immune dysregulation affects approximately 39 patients per million globally and currently has no approved therapies, making this trial particularly significant for addressing a substantial unmet medical need.

MHRA Approves Joenja (leniolisib) as First Treatment for Rare Immune Disease APDS

• The MHRA has approved Joenja (leniolisib) as the first medicine for Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS) in the UK. • APDS is a rare, inherited immune disorder affecting approximately 30-40 people in the UK, characterized by recurrent infections and abnormal immune function. • Leniolisib demonstrated a statistically significant reduction in lymph node size and normalization of immunophenotype in a 12-week placebo-controlled trial. • The approval was expedited through the International Recognition Procedure (IRP), leveraging prior FDA approval to provide quicker access to UK patients.
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