Top Qs
Timeline
Chat
Perspective
Leniolisib
Medication From Wikipedia, the free encyclopedia
Remove ads
Leniolisib (INN[3][4]), sold under the brand name Joenja, is a medication used for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS).[2][5] It is a kinase inhibitor[2][6] that is taken by mouth.[2]
Remove ads
The most common side effects include headache, sinusitis, and atopic dermatitis.[5]
Leniolisib was approved for medical use in the United States in March 2023.[5][7][8] It is the first approved medication for the treatment of activated PI3K delta syndrome.[5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[9]
Remove ads
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.[2][5]
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.[5] 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.[5]
Remove ads
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.[5]
Remove ads
Adverse effects
The most common encountered adverse effects were headache, sinusitis, and atopic dermatitis.[2]
History
Leniolisib was developed by Novartis and subsequently licensed to Pharming Group, a Dutch biotechnology company, in 2019.[7]
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.[5]
The FDA granted the application for leniolisib orphan drug, priority review, and rare pediatric disease designations.[5]
Remove ads
References
External links
Wikiwand - on
Seamless Wikipedia browsing. On steroids.
Remove ads