Former Novartis drug for ultra-rare disease gets speedy FDA review

Three years after paying a modest $20 million upfront to grab a late-stage rare disease drug from Novartis, Pharming has lined up a priority review at the FDA.

Known at the time as CDZ173, leniolisib belongs to the PI3Kẟ (phosphoinositide 3-kinase delta) inhibitor class, which is best known for cancer applications. But Novartis took a different path, testing it for an immunodeficiency disease called activated phosphoinositide 3-kinase delta syndrome, or APDS.