Pharming Group Receives FDA Complete Response Letter For Joenja Pediatric SNDA

India, Feb. 1 -- Pharming Group (PHAR) 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). Joenja is an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor being developed as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency.

The FDA raised concerns regarding the potential for underexposure in lower-weight pediatric patients. To address this, the Agency has requested additional pediatric pharmacokinetic data to reassess the proposed dosing regimen and confirm that children in the lower weight groups can achieve expo...