FDA approves leniolisib for activated phosphoinositide 3-kinase delta syndrome

The FDA approved leniolisib as the first treatment for activated phosphoinositide 3-kinase delta syndrome in adults and pediatric patients 12 years of age and older.

According to a press release from the FDA, leniolisib (Joenja, Pharming Group)was evaluated for efficacy in a 12week blinded, randomized, placebo-controlled study of 31 adult and pediatric patients 12 years of age and older with confirmed activated phosphoinositide 3-kinase delta syndrome (APDS)-associated genetic PI3Kd mutation, with a documented variant in either PIK3CD or PIK3R1. Researchers considered improvement in lymphoproliferation and the normalization of immunophenotype as measured by the percentage of naïve B cells out of total B cells as the co-primary efficacy endpoints.

The release noted 21 patients received 70 mg of leniolisib and 10 patients received placebo twice a day for 12 weeks. Patients in the leniolisib group had a reduction in lymph node size and a 37% improvement in naïve B cell counts by day 85. The most common side effects of the drug included headache, atopic dermatitis and sinusitis, according to the release.

Leniolisib previously received priority review for APDS in 2022.