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.

Pharming Group N.V.

Pharming Group N.V. (Euronext Amsterdam: PHARM/Nasdaq: PHAR) is a global biopharmaceutical company dedicated to transforming the lives of patients with rare, debilitating, and life-threatening diseases. Pharming is commercializing and developing an innovative portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development. Pharming is headquartered in Leiden, Netherlands, and has employees around the globe who serve patients in over 30 markets in North America, Europe, the Middle East, Africa, and Asia-Pacific. For more information, visit www.pharming.com.

28 March, 2023

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