JOENJA

What it is

JOENJA is a film-coated tablet containing leniolisib phosphate equivalent to 70 mg leniolisib. Leniolisib selectively inhibits phosphoinositide 3-kinase delta (PI3Kδ) by blocking the active binding site of PI3Kδ. Gain-of-function variants in the gene encoding the p110δ catalytic subunit or loss-of-function variants in the p85α regulatory subunit lead to hyperactive PI3Kδ signalling, and leniolisib reduces this hyperactivity by decreasing production of PIP3, thereby modifying dysregulation of B and T cell populations and disease progression. JOENJA is subject to additional monitoring in Australia.

Approved indications

— Activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older.

Dosing overview

The recommended dose in adult and paediatric patients who weigh more than 45 kg is 70 mg JOENJA twice daily approximately 12 hours apart. The film coated tablet can be taken with or without food.

Key safety warnings

JOENJA may cause fetal harm when administered to a pregnant woman, as administration of leniolisib to rats and rabbits during organogenesis caused embryo-fetal toxicity including malformations at exposures that were 2 to 6 times higher than the recommended human dose. Females of reproductive potential should use highly effective methods of contraception during treatment and for 1 week after the last dose. Serious, sometimes fatal, immune-related adverse events such as severe infections, severe cutaneous adverse reactions, pneumonitis, severe diarrhoea/colitis potentially resulting in intestinal perforation, neutropenia, and hepatotoxicity have occurred in patients receiving other PI3K delta inhibitors for the treatment of haematological or solid cancers. While these serious events have not been associated with the use of JOENJA in APDS patients, the safety data for JOENJA is limited. Live, attenuated vaccinations may be less effective if administered during JOENJA treatment. JOENJA has been associated with decreases in neutrophil counts.

Contraindications

Hypersensitivity to the active substance or to any of the excipients. Patients with rare hereditary problems of galactose intolerance, total lactase deficiency, or glucose-galactose malabsorption must not take this medicine.

Regulatory history

JOENJA leniolisib 70 mg film coated tablets was first registered on the ARTG on 18 March 2025. In July 2025, the PBAC recommended JOENJA for symptomatic activated phosphoinositide 3-kinase delta syndrome in adults and adolescents aged 12 years and older.