Leniolisib for treating activated phosphoinositide 3- kinase delta Syndrome in children aged 4 to 11 years

Leniolisib is in clinical development for paediatric patients aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS). APDS is an inherited rare disorder caused by
defects in the genes that control the production of a protein called phosphoinositide 3-kinase delta (PI3K delta). These defects lead to PI3K delta becoming overactive which alters the
development and control of immune cells (B and T cells) and their ability to fight off infections. Beginning in childhood, people with APDS develop recurrent infections, particularly in the lungs, sinuses, and ears, which over time, damages the passages leading from the windpipe to the lungs (bronchi) and can cause breathing problems. Currently, treatment of APDS in children is limited to supportive therapies or, if considered appropriate, a bone marrow transplant. Therefore, there is an unmet need for targeted treatment options for APDS in younger patients.