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Category: activated PI3K delta syndrome

  • Pharming preps filing for rare disease drug leniolisib after trial win

    Pharming has taken a step closer to getting a second product to market, after its rare disease therapy leniolisib met its objectives in a pivotal trial, setting up regulatory filings in the coming months. The Netherlands biotech is developing leniolisib (formerly CDZ173) under license from Novartis for activated PI3K delta syndrome (APDS), an ultra-rare disease […]