Pharming announces US FDA approval of Joenja? (leniolisib) as the first and only treatment indicated for APDS

發(fā)布時間： 2023-04-10 閱讀：545次

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March 24, 2023 -- Pharming Group N.V. (“Pharming” or “the Company”)

(EURONEXT Amsterdam: PHARM/Nasdaq: PHAR) announces that the US Food and Drug Administration (FDA) has approved Joenja? (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Joenja?, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the US for APDS, a rare and progressive primary immunodeficiency. The FDA evaluated the Joenja? application for APDS under Priority Review, which is granted to therapies that have the potential to provide significant improvements in the treatment, diagnosis or prevention of serious conditions. Joenja? is expected to launch in the US in early April and will be available for shipment in mid-April.

Dr. Eveline Wu, MD, MSCR, Division Chief, Paediatric Rheumatology & Associate Professor of Paediatric Rheumatology and Allergy/Immunology at The University of North Carolina School of Medicine, said: “The FDA approval of Joenja? is an exciting moment for the APDS community and offers to transform the treatment pathway for patients and families affected by this rare disease. This approval means that they will, for the first time, have access to an approved treatment, which has the potential to change the standard of care for the patient population suffering from APDS.”

The FDA evaluated the New Drug Application (NDA) for Joenja? under priority review and has approved the drug based on findings from a multinational, triple-blind, placebo-controlled, randomized Phase II/III clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS aged 12 years and older. Also submitted as part of the application were data from a longterm, open-label extension clinical trial in which 38 patients received Joenja? for a median of two years.

Results from the 12-week randomized, placebo-controlled study in 31 patients with APDS aged 12 years and older demonstrated clinical efficacy of Joenja? 70mg twice daily over placebo, and was significant in the co-primary endpoints which evaluated improvement in lymphoproliferation as measured by the reduction in lymph node size and increase in na?ve B cells, reflecting the impact on immune dysregulation and normalization of immunophenotype in these patients, respectively.