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FDA Alerts
Hematology

FDA approves leniolisib for activated phosphoinositide 3-kinase delta syndrome

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The US Food and Drug Administration has approved leniolisib (Joenja; Pharming Group) for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in patients aged 12 and over, according to a press release.

APDS is a rare primary immunodeficiency, affecting an estimated 1 to 2 people per million. Joenja is the first and only treatment to be approved in the US for the condition, and it is expected to launch in early April. The FDA evaluated the new drug application for Joenja 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.

The approval was based on the findings from a multinational, triple-blind, placebo-controlled, randomized Phase 2/3 clinical trial, which evaluated efficacy and safety in 31 patients diagnosed with APDS. Results from the study showed that leniolisib 70mg twice daily was significantly effective in reducing lymph node size and increasing naïve B cells, reflecting the impact on immune dysregulation and normalization of immunophenotype in these patients. The drug was well-tolerated with the most common adverse reactions being headache, sinusitis, and atopic dermatitis.

Read the full press release here.

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