Clinical data | |
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Trade names | Joenja |
Other names | CDZ173 |
AHFS/ Drugs.com | Monograph |
MedlinePlus | a623016 |
License data |
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Routes of administration | By mouth |
Drug class | Antineoplastic |
ATC code | |
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Identifiers | |
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PDB ligand | |
Chemical and physical data | |
Formula | C21H25F3N6O2 |
Molar mass | 450.466 g·mol−1 |
3D model ( JSmol) | |
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Leniolisib (INN [2] [3]), sold under the brand name Joenja, is a medication used for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS). [1] [4] It is a kinase inhibitor [1] [5] that is taken by mouth. [1]
The most common side effects include headache, sinusitis, and atopic dermatitis. [4]
Leniolisib was approved for medical use in the United States in March 2023. [4] [6] [7] It is the first approved medication for the treatment of activated PI3K delta syndrome. [4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [8]
Leniolisib is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (activated PI3K delta syndrome) in people twelve years of age and older. [1] [4]
Activated PI3K delta syndrome is caused by mutations in either of two genes, PIK3CD or PIK3R1, that regulate the maturation of white blood cells, especially B cells and T cells. [4] This leads to a decrease in immune cells, which makes it difficult for people with activated PI3K delta syndrome to fight off bacterial and viral infections. [4]
Leniolisib is a selective phosphoinositide 3-kinase inhibitor (PI3Kδ inhibitor), which means it blocks a form of the protein called phosphoinositide 3-kinase delta (PI3Kδ) that is overactive in activated PI3K delta syndrome. By inhibiting PI3Kδ, leniolisib helps normalize immune function as measured by a significant increase in number of immune response generating B cells and reduction in size of lymph nodes. [4]
The most common encountered adverse effects were headache, sinusitis, and atopic dermatitis. [1]
Leniolisib was developed by Novartis and subsequently licensed to Pharming Group, a Dutch biotechnology company, in 2019. [6]
The US Food and Drug Administration (FDA) evaluated the efficacy of leniolisib in the placebo-controlled portion of Study 2201 (NCT02435173), a twelve‑week blinded, randomized, placebo-controlled study of 31 participants twelve years of age and older with confirmed APDS-associated genetic PI3Kδ mutation, with a documented variant in either PIK3CD or PIK3R1. [4]
The FDA granted the application for leniolisib orphan drug, priority review, and rare pediatric disease designations. [4]
This article incorporates text from this source, which is in the public domain: Bing Chat output modified to create the initial revision of this article. 25 March 2023. – via Microsoft