The FDA has issued a Complete Response Letter (CRL) to Pharming Group to its supplemental New Drug Application (sNDA) for Joenja (leniolisib) for patients aged four to 11 with activated phosphoinositide 3-kinase delta syndrome (APDS).
Leniolisib is an oral, selective phosphoinositide 3-kinase delta inhibitor that is approved for the treatment of adults and children aged 12 and above with APDS, a rare primary immunodeficiency. The FDA issued the CRL to the sNDA for younger patients due to concerns that there may be potential for underexposure in patients of a lower weight. The FDA has asked Pharming Group to provide additional pediatric pharmacokinetic data to confirm that children in the lower weight dose groups can achieve exposure levels comparable to the approved adult and adolescent regimen.
The CRL also requests additional data and clarification around one of the analytical methods used for production batch testing. Pharming Group has said it will be asking the FDA for a Type A meeting to determine the steps for resubmission.