Pharming Group Receives Complete Response Letter from FDA for Joenja Application

Pharming Group 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, an oral, selective phosphoinositide 3-kinase delta 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 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.