Sijmen de Vries, Pharming CEO

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.

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