FDA Approves First-Ever Treatment for Activated Phosphoinositide 3-Kinase Delta Syndrome

The FDA has approved Pharming’s Joenja (leniolisib) twice-daily tablets as the first treatment for adult and pediatric patients 12 years and older with activated phosphoinositide 3-kinase delta syndrome, a rare genetic disorder that causes low white blood cell counts.

Potential symptoms of the disorder also include recurrent infections and enlarged lymph nodes, as well as a higher risk of developing blood cancers.

Joenja is a selective phosphoinositide 3-kinase delta inhibitor, a type of drug that has anticancer properties and potential effects on lymphomas.

Pharming gained exclusive rights to the drug from Novartis under a collaboration and licensing deal inked in August 2019.

The FDA previously granted Joenja Orphan Drug and Rare Pediatric Disease designations as well as a priority review.