Pharming receives positive recommendation from NICE for Joenja®▼(leniolisib) as a treatment for APDS

Core Viewpoint - Pharming Group N.V. has received positive final guidance from the National Institute for Health and Care Excellence (NICE) for the reimbursement and use of Joenja® (leniolisib) in treating activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in patients aged 12 and older within the NHS in England and Wales [1][2][4] Group 1: NICE Recommendation and Clinical Data - The NICE recommendation is based on comprehensive data from a Phase III clinical trial that showed significant improvements in immune deficiency and dysregulation with leniolisib compared to placebo [2][4] - The Phase III trial results indicated sustained clinical improvements during long-term treatment in an open-label extension trial [2] Group 2: Impact on Patients and Healthcare - The NICE recommendation is viewed as a significant milestone for patients with APDS, enhancing treatment options and potentially improving their quality of life [3][4] - Joenja® is now available for use and funded in England through the Innovative Medicines Fund, with expected funding in Wales within three months [4] Group 3: About Joenja® and APDS - Joenja® is the first and only targeted treatment for APDS, an oral small molecule PI3Kδ inhibitor that addresses the underlying immune system issues associated with the condition [8] - APDS is a rare primary immunodeficiency affecting approximately 1 to 2 people per million worldwide, characterized by severe recurrent infections and immune dysregulation [6][7]