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Pharming Reports the US FDA’s sNDA Acceptance and Priority Review to Leniolisib for Activated Phosphoinositide 3-Kinase Delta Syndrome

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  • The US FDA has accepted sNDA & granted priority review to leniolisib for the treatment of children (4-11yrs.) with activated phosphoinositide 3-kinase delta (APKD) syndrome (PDUFA: Jan 31, 2026); regulatory review is ongoing in the EEA, Japan & Canada
  • sNDA was supported by a P-III trial in APKD pediatric pts (4-11yrs.), which showed reduced lymphadenopathy & increased naïve B cells over 12wks. & was further backed by safety data from 8mos. of treatment
  • Leniolisib (PO), a PI3Kẟ inhibitor marketed under the brand name Joenja in the US, is also being investigated in 2 P-III trials for APDS in children & 2 P-II trials for primary immunodeficiencies with immune dysregulation

Ref: Pharming | Image: Pharming | Press Release

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