Research & Development
Pharming's leniolisib to treat rare immunodeficiency granted accelerated assessment for MAA in Europe
1 August 2022 -

Biopharmaceutical company Pharming Group N.V. (Euronext Amsterdam:PHARM) (Nasdaq:PHAR) announced on Monday that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted an accelerated assessment for the Marketing Authorisation Application (MAA) for leniolisib.

The company said leniolisib has been studied for the treatment of activated PI3K delta syndrome (APDS), a rare primary immunodeficiency, in adults and adolescents age 12 or older in the European Economic Area (EEA). It added that it is on track and plans to submit its MAA for leniolisib to the EMA in October 2022.

Clinical development for leniolisib includes positive data from a Phase II/III study of the product, which met both its co-primary endpoints in the target patient population of evaluated reduction in lymph node size and correction of immunodeficiency. In this study, leniolisib was generally well-tolerated, with the majority of reported adverse events in both treatment groups classified as mild.

Pharming said it is dedicated to transforming the lives of patients with rare, debilitating and life-threatening diseases. It is commercialising and developing a portfolio of protein replacement therapies and precision medicines, including small molecules, biologics, and gene therapies that are in early to late-stage development.



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