FDA seeks more Joenja data for children 4-11 with rare immune disorder

Pharming Group receives Complete Response Letter from U.S. FDA for sNDA for Joenja (leniolisib) in children aged 4 to 11 years with APDS

Leiden, the Netherlands, February 1, 2026: Pharming Group (Euronext: PHARM; Nasdaq: PHAR) today announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to its supplemental New Drug Application (sNDA) for Joenja® (leniolisib), an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, as a treatment for children aged 4 to […] The post Pharming Group receives Complete Response Letter from U…

FDA seeks more Joenja data for children 4-11 with rare immune disorder

With no approved APDS treatments for children under 12, FDA's CRL on Pharming's Joenja sNDA demands more pediatric data before dose reassessment.