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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 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency. The FDA raise
About this update from Pharming Group Nv
[{"type":"image","alt":"Pharming Group N.V.","displaySize":"","headline":null,"caption":"Pharming Group N.V.","className":"","disableSlideshowImg":false,"size":{"original":{"width":300,"height":102,"url":"https://media.zenfs.com/en/globenewswire.com/e33000c1e8641d4ca14a349a419f6745"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/6VI_P53XoKPQr35Rp9bMIg--/YXBwaWQ9aGlnaGxhbmRlcjt3PTQyMDtoPTE0MztjZj13ZWJw/https://media.zenfs.com/en/globenewswire.com/e33000c1e8641d4ca14a349a419f6745","width":300,"height":102}},"lazy":false},{"type":"text","content":"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 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.","length":486,"tagName":"p"},{"type":"text","content":"The FDA raised an issue with the potential for underexposure in lower weight pediatric patients. As a result, the FDA has requested additional pediatric pharmacokinetic data to reassess the proposed pediatric doses and confirm that children in the lower weight dose groups can achieve exposure levels comparable to the approved adult and adolescent regimen. The letter also identified an issue with one of the analytical methods used for production batch testing, and the FDA requested additional data and clarification on this point.","length":534,"tagName":"p"},{"type":"text","content":"We believe we can address the clinical pharmacology and batch testing methodology issues outlined in the letter, and we plan to work closely with the FDA to meet the Agency’s requirements and determine next steps for resubmission. We plan to request a Type A meeting with the FDA.","length":280,"tagName":"p"},{"type":"text","content":"Joenja’s U.S. FDA approval for the treatment of APDS in patients aged 12 years of age and older is unaffected by this regulatory action.","length":136,"tagName":"p"},{"type":"text","content":"Fabrice Chouraqui, Chief Executive Officer of Pharming, commented:","length":66,"tagName":"p"},{"type":"text","content":"“While we are disappointed in the FDA’s response, we remain...