Leniolisib
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] Leniolisib is a kinase inhibitor.[1]
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| Trade names | Joenja |
| Other names | CDZ173 |
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| Routes of administration | By mouth |
| Drug class | Antineoplastic |
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| Formula | C21H25F3N6O2 |
| Molar mass | 450.466 g·mol−1 |
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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][5][6] It is the first approved medication for the treatment of activated PI3K delta syndrome.[4]
Medical uses
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]
Mechanism of action
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]
Adverse effects
The most common encountered adverse effects were headache, sinusitis, and atopic dermatitis.[1]
History
Leniolisib was developed by Novartis and subsequently licensed to Pharming Group, a Dutch biotechnology company, in 2019.[5]
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]
References
- "Joenja- leniolisib tablet, film coated". DailyMed. 29 March 2023. Retrieved 20 June 2023.
- World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 76". WHO Drug Information. 30 (3). hdl:10665/331020.
- World Health Organization (2017). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 77". WHO Drug Information. 31 (1). hdl:10665/330984.
- "FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome". U.S. Food and Drug Administration (FDA) (Press release). 24 March 2023. Retrieved 24 March 2023.
This article incorporates text from this source, which is in the public domain. - "US FDA approves Pharming's immune disorder drug". Reuters. Archived from the original on 24 March 2023. Retrieved 24 March 2023.
- "Pharming announces US FDA approval of Joenja (leniolisib) as the first and only treatment indicated for APDS" (PDF). Pharming Group N.V. (Press release). 24 March 2023. Retrieved 25 March 2023.
This article incorporates text generated in part with Bing by OpenAI. The author of each LLM-assisted edit takes ultimate responsibility for the added content; see the page's history for attribution.
External links
- Clinical trial number NCT02435173 for "Study of Efficacy of CDZ173 in Patients With APDS/PASLI" at ClinicalTrials.gov