|Drug class||Kinase inhibitor|
|Main uses||Activated phosphoinositide 3-kinase delta syndrome (APDS)|
|Side effects||Headache, sinusitis, atopic dermatitis|
|Typical dose||70 mg BID|
|Chemical and physical data|
|Molar mass||450.466 g·mol−1|
|3D model (JSmol)|
Leniolisib, sold under the brand name Joenja, is a medication used to treat activated phosphoinositide 3-kinase delta syndrome (APDS). It is used in people at least 12 years old. It is taken by mouth.
Common side effects include headache, sinusitis, and atopic dermatitis. Use in pregnancy or breastfeeding may harm the baby. It should not be used in people with significant liver problems. It is a kinase inhibitor.
Leniolisib was approved for medical use in the United States in 2023. It received an orphan medication designation in Europe in 2020. In the United States it costs about 550,000 USD per year as of 2023. It is the first medication for APDS.
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. 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.
In those who weight more than 45 kg the dose is 70 mg twice per day.
The most common side effects were headache, sinusitis, and atopic dermatitis.
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.
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.
Society and culture
- "Joenja- leniolisib tablet, film coated". DailyMed. 29 March 2023. Archived from the original on 1 July 2023. Retrieved 20 June 2023.
- "EU/3/20/2339". European Medicines Agency. 12 February 2021. Archived from the original on 8 July 2022. Retrieved 22 June 2023.
- "Leniolisib Prices, Coupons & Savings Tips - GoodRx". www.goodrx.com. Retrieved 22 June 2023.
- "FDA approves first treatment for activated phosphoinositide 3-kinase delta syndrome". U.S. Food and Drug Administration (FDA) (Press release). 24 March 2023. Archived from the original on 25 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.
- 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.