FDA broadens APDS treatment options

An 8-patient study evaluated safety and pharmacokinetics in the younger pediatric population.

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The FDA has approved leniolisib (Joenja) for treating activated phosphoinositide 3-kinase delta syndrome in pediatric patients aged 4 to 11 years.
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Leniolisib was initially approved for patients aged 12 years and older in 2023, targeting a rare genetic disorder affecting the immune system.
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APDS can cause recurrent infections and enlargement of lymph nodes, tonsils, and spleen, potentially leading to airway and gastrointestinal obstruction.
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Efficacy for older patients was demonstrated in a 12-week study showing reduced lymph node size and improved naïve B-cell counts with leniolisib.
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Common adverse effects in younger patients include abdominal pain, respiratory infections, diarrhea, and headache; leniolisib is contraindicated in severe liver impairment.

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