The MAA was granted based on the data from the P-II/III study evaluating the safety & efficacy of leniolisib in adolescents & adults (≥12yrs.) with APDS. The MAA submission was also supported by the data from an extension clinical trial evaluating leniolisib for the same indication
The results demonstrated a reduction in lymph node size along with an increase in the percentage of naïve B cells in patients with APDS. Additionally, the safety data depicted that leniolisib was well tolerated by all patients
Leniolisib is a small molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K, that functions by inhibiting the production of PIP3 which is a cellular messenger activating AKT & cell function regulator
Ref: PR Newswire | Image: Pharming
