leniolisib leniolisib leniolisib, sold under the brand name joenja, is a medication used for the treatment of activated phosphoinositide 3-kinase delta syndrome (apds). leniolisib is a kinase inhibitor. leniolisibclinical datatrade namesjoenjaother namescdz173license data us dailymed: leniolisib routes ofadministrationby mouthdrug classantineoplasticatc codenonelegal statuslegal status us: ℞-only identifierscas number1354690-24-6as salt: 1354691-97-6drugbankdb16217chemspider52083264uniil22772z9cpkeggd11158as salt: d11159chemblchembl3643413pdb ligand9nq (pdbe, rcsb pdb)chemical and physical dataformulac21h25f3n6o2molar mass450.466 g·mol−13d model (jsmol)interactive image smiles ccc(=o)n1cc(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/s1key:mwkymzxcgyxlpl-zdusscgksa-n the most common side effects include headache, sinusitis, and atopic dermatitis. leniolisib was approved for medical use in the united states in march 2023. it is the first approved drug for this indication. medical uses leniolisib is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (apds) in people twelve years of age and older. apds 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 apds to fight off bacterial and viral infections. mechanism of action leniolisib is an oral, selective pi3kδ inhibitor, which means it blocks a form of the protein called phosphoinositide 3-kinase delta (pi3kδ) that is overactive in apds. 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. adverse effects the most common encountered adverse effects were headache, sinusitis, and atopic dermatitis. history leniolisib was originally developed by novartis and subsequently licensed globally to pharming group, a dutch biotechnology company, in 2019. the u.s. 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. the fda granted the application for leniolisib orphan drug, priority review, rare pediatric disease designations. society and culture names leniolisib is the international nonproprietary name.