Pharming’s Joenja (leniolisib) Receives the US FDA’s Approval as First Treatment for Activated Phosphoinositide 3-Kinase Delta Syndrome
- The US FDA has approved Joenja (selective PI3Kδ inhibitor) for APDS in adult & pediatric patients aged ≥12yrs. The product is expected to be available in the US in early April & will be available for shipment in mid-April
- The approval was based on the P-II/III trial evaluating Joenja (70mg, BID) vs PBO in 31 patients. The 12wk. results showed the clinical efficacy of Joenja & were significant in the co-1EPs that assessed improvement in lymphoproliferation as measured by a decrease in lymph node size & increase in naïve B cells thus reflecting the impact on immune dysregulation & normalization of immunophenotype
- The MAA of leniolisib is under the EMA’s review & opinion is expected in H2’23. The company also plans for regulatory approvals in the UK, Canada, Australia & Japan
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