Leniolisib

Leniolisib
Names
Trade namesJoenja
Other namesCDZ173
Clinical data
Drug classKinase inhibitor[1]
Main usesActivated phosphoinositide 3-kinase delta syndrome (APDS)[1]
Side effectsHeadache, sinusitis, atopic dermatitis[1]
WHO AWaReUnlinkedWikibase error: ⧼unlinkedwikibase-error-statements-entity-not-set⧽
Pregnancy
category
  • May cause harm[1]
BreastfeedingNot recommended[1]
Routes of
use		By mouth[1]
Typical dose70 mg BID[1]
Legal
License data
Legal status
Chemical and physical data
FormulaC21H25F3N6O2
Molar mass450.466 g·mol−1
3D model (JSmol)
SMILES
  • CCC(=O)N1CC[C@@H](C1)NC1=C2CN(CCC2=NC=N1)C1=CN=C(OC)C(=C1)C(F)(F)F
InChI
  • InChI=1S/C21H25F3N6O2/c1-3-18(31)30-6-4-13(10-30)28-19-15-11-29(7-5-17(15)26-12-27-19)14-8-16(21(22,23)24)20(32-2)25-9-14/h8-9,12-13H,3-7,10-11H2,1-2H3,(H,26,27,28)/t13-/m0/s1
  • Key:MWKYMZXCGYXLPL-ZDUSSCGKSA-N

Leniolisib, sold under the brand name Joenja, is a medication used to treat activated phosphoinositide 3-kinase delta syndrome (APDS).[1] It is used in people at least 12 years old.[1] It is taken by mouth.[1]

Common side effects include headache, sinusitis, and atopic dermatitis.[1] Use in pregnancy or breastfeeding may harm the baby.[1] It should not be used in people with significant liver problems.[1] It is a kinase inhibitor.[1]

Leniolisib was approved for medical use in the United States in 2023.[1] It received an orphan medication designation in Europe in 2020.[2] In the United States it costs about 550,000 USD per year as of 2023.[3] It is the first medication for APDS.[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]

Dosage

In those who weight more than 45 kg the dose is 70 mg twice per day.[1]

Side effects

The most common side effects were headache, sinusitis, and atopic dermatitis.[1]

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]

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]

Society and culture

Leniolisib is the INN.[6][7]

References

  1. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 16 17 18 19 "Joenja- leniolisib tablet, film coated". DailyMed. 29 March 2023. Archived from the original on 1 July 2023. Retrieved 20 June 2023.
  2. ↑ "EU/3/20/2339". European Medicines Agency. 12 February 2021. Archived from the original on 8 July 2022. Retrieved 22 June 2023.
  3. ↑ "Leniolisib Prices, Coupons & Savings Tips - GoodRx". www.goodrx.com. Retrieved 22 June 2023.
  4. 1 2 3 4 5 6 7 "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. Public Domain This article incorporates text from this source, which is in the public domain.
  5. ↑ "US FDA approves Pharming's immune disorder drug". Reuters. Archived from the original on 24 March 2023. Retrieved 24 March 2023.
  6. ↑ World Health Organization (2016). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 76". WHO Drug Information. 30 (3). hdl:10665/331020.
  7. ↑ World Health Organization (2017). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 77". WHO Drug Information. 31 (1). hdl:10665/330984.
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