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Components of U.S. Deferred Tax Assets and (liabilities) (Detail) - USD ($)
$ in Thousands
Dec. 31, 2018
Dec. 31, 2017
Deferred tax assets:    
Federal and state net operating loss carryforwards $ 241,881 $ 203,897
Federal and state research tax credit carryforwards 23,917 21,238
Federal Orphan Drug Credit 120,273 96,750
Deferred revenue 29,530 17,538
Stock options 19,992 15,995
Other 9,448 7,529
Net deferred tax assets before valuation allowance 445,041 362,947
Valuation allowance $ (445,041) $ (362,947)