FDA has recently approved leniolisib tablets as the first treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in patients aged 12 years and above. APDS is caused by mutations in phosphoinositide-3 kinase genes, PIK3CD or PIK3R1, resulting in immune system impairment by lowering the numbers of white blood cells, especially B and T cells. The approval of leniolisib is based on a clinical trial conducted on APDS patients.
Clinical trial In the randomized, 12-week blinded, placebo-controlled study, 31 adults and pediatric (aged 12 years and above) patients were treated with 70 mg of leniolisib or placebo twice daily for 12 weeks. Leniolisib reduced lymph node size and improved naïve B cell counts by 37% compared to the placebo at day 85 of the study. Recommended dosage 70 mg orally twice a day, 12 hours apart, for patients weighing at least 45 kg Adverse reactions…
