General Information

Joenja (leniolisib) is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor.

Joenja is specifically indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

Joenja is supplied as tablets for oral administration. The recommended dosage in adult and pediatric patients 12 years of age and older weighing 45 kg or greater is 70 mg administered orally twice daily approximately 12 hours apart, with or without food. There is no recommended dosage for patients weighing less than 45 kg.

Advise patients that if a dose is missed by more than 6 hours, wait and take the next dose at the usual time.

 Advise patients that if vomiting occurs within 1 hour after taking Joenja , take the next dose as soon as possible. If vomiting occurs more than 1 hour after dosing, wait and take the next dose at the usual time.

Mechanism of Action

Joenja (leniolisib) is an oral small molecule phosphoinositide 3-kinase delta (PI3Kẟ) inhibitor. Joenja inhibits the production of phosphatidylinositol-3-4-5-trisphosphate, which serves as an important cellular messenger and regulates a multitude of cell functions including proliferation, differentiation, cytokine production, cell survival, angiogenesis, and metabolism.

Side Effects

Adverse effects associated with the use of Joenja may include, but are not limited to, the following:

• headache
• sinusitis
• atopic dermatitis

Clinical Trial Results

FDA approval was based on findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older. Results from the 12-week study demonstrated clinical efficacy of Joenja 70mg twice daily over placebo, and was significant in the co-primary endpoints which evaluated improvement in lymphoproliferation as measured by the reduction in lymph node size and increase in naïve B cells, reflecting the impact on immune dysregulation and normalization of immunophenotype in these patients, respectively. The adjusted mean change (95% CI) between Joenja and placebo for lymph node size was -0.25 (–0.38,-0.12; P=0.0006; N=26) and for percentage of naïve B cells was 37.30 (24.06, 50.54; P=0.0002; N=13).