NICE (UK) recommends Joenja (leniolisib) for treating activated phosphoinositide 3-kinase delta syndrome in people 12 years and over – Pharming

NICE (UK): Leniolisib is recommended, within its marketing authorisation, for treating activated phosphoinositide 3‑kinase delta syndrome (APDS) in people 12 years and over. Leniolisib is recommended only if the company provides it according to the commercial arrangement.

Why the committee made this recommendation: APDS is an ultra-rare genetic condition that can severely affect the quality of life of people with the condition, and their families and carers, and can significantly shorten life. It can cause organs and lymph nodes to swell and the body’s immune system to attack healthy tissue. People with the condition are also at high risk of serious infections, and it can increase the risk of cancer. There are no licensed treatments for APDS. Standard care includes antimicrobial treatment, surgery, immunosuppressants, immunoglobulin (antibody) replacement therapy, and stem cell transplants.

Clinical trial evidence shows that leniolisib, compared with placebo plus selected standard care treatments, reduces the size of people’s lymph nodes and increases levels of immune cells called B cells. The cost-effectiveness estimates are within the range that NICE considers an acceptable use of NHS resources. So, leniolisib is recommended.