(RTTNews) - Pharming Group N.V. (PHAR) announced that the U.S. Food and Drug Administration has accepted its supplemental New Drug Application for leniolisib, an oral, selective PI3Kd inhibitor, as a treatment for children aged 4 to 11 with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The application received Priority Review and a PDUFA target action date of January 31, 2026.
The supplemental New Drug Application (sNDA) submitted to the FDA is supported by positive results from a multinational, single-arm Phase III study in children aged 4 to 11 years. Over a 12-week period, the study demonstrated improvements in two key indicators of activated phosphoinositide 3-kinase delta syndrome (APDS): reduced lymphadenopathy and increased levels of naïve B cells, suggesting a correction of the underlying immune dysfunction. The application also includes safety data from eight months of treatment.
Leniolisib, marketed under the brand name Joenja in the U.S., received approval from the U.S. FDA for the treatment of APDS in adult and pediatric patients 12 years of age and older in March 2023.
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