NICE recommends Pharming’s Joenja as treatment for APDS

The UK’s National Institute for Health and Care Excellence (NICE) has issued positive final guidance recommending Joenja (leniolisib), from Dutch biopharma company Pharming (Nasdaq: PHAR), for reimbursement and use within the National Health Service (NHS) in England and Wales for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

The health technology assessor’s recommendation is based on the totality of the data, including the Phase III clinical trial evaluating leniolisib, an oral selective PI3Kδ inhibitor, in patients with APDS, a rare and progressive primary immunodeficiency. In the primary analysis, treatment with leniolisib led to significant improvements in immune deficiency and immune dysregulation compared to placebo. The Phase III trial demonstrated an improvement in clinical outcomes, which was maintained during longer-term treatment within the open-label extension trial.

“This positive recommendation for Joenja® from NICE is a significant milestone for patients living with APDS in England and Wales. It underscores Pharming’s commitment to work closely with regulatory and reimbursement authorities, the medical immunology community, and patient associations to bring this first targeted treatment to patients who may benefit,” commented Pharming’s chief executive Fabrice Chouraqui.