The U.S. Food and Drug Administration (FDA) on Friday issued a Complete Response Letter (CRL) to Pharming Group’s (NASDAQ:PHAR) supplemental New Drug Application (sNDA) for Joenja (leniolisib) for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.
APDS causes recurrent respiratory infections, chronic viral infections, and immune dysfunction, including lymphoproliferation (enlarged spleen/lymph nodes), autoimmunity, and increased lymphoma risk.
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.
Why It Matters
The company on Friday said it plans to request a Type A meeting with the FDA.
Joenja’s U.S. FDA approval (in March 2023) for APDS in patients aged 12 years of age and older is unaffected by this regulatory action.
Pharming submitted the sNDA to the FDA based on data from the open-label, multinational, single-arm Phase 3 study in children aged 4 to 11 years, which showed improvements over 12 weeks in two clinically relevant hallmarks of APDS, reduced lymphadenopathy and increased naïve B cells, together indicating a correction of the underlying immune defect.
The submission also included safety data from 8 months of treatment.
The improvements in lymphoproliferation and immunophenotype correction were seen across the four dose levels investigated and were consistent with the improvements previously reported in adolescent and adult patients.
PHAR Price Action: Pharming shares were down 16.76% at $17.03 during premarket trading on Monday, according to Benzinga Pro data.
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