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
- -
MHRA approves Pharming's Joenja as first drug for rare immune disease APDS
MHRA approves Pharming Group’s Joenja (leniolisib) for activated phosphoinositide 3-kinase delta syndrome (APDS), the first drug for this rare immune disease in the UK. Indicated for adults and adolescents aged 12+ weighing at least 45kg, Joenja reduces lymph node size and improves B cell counts. Approval via the International Recognition Procedure (IRP) was supported by a 12-week study.
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