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CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($)
$ in Thousands
 3 Months Ended 9 Months Ended
Sep. 30, 2023
Sep. 30, 2022
Sep. 30, 2023
Sep. 30, 2022
Product revenue, net $ 22,504 $ 4,284 $ 53,194 $ 14,848
Costs and expenses:        
Cost of sales 2,412 497 6,069 1,105
Research and development 33,570 21,116 105,059 85,082
Selling, general, and administrative 30,475 21,938 85,158 65,715
Total costs and expenses 66,457 43,551 196,286 151,902
Loss from operations (43,953) (39,267) (143,092) (137,054)
Other income (expense):        
Other income (expense), net (159) (370) (369) (370)
Interest expense (3,149) (2,144) (9,342) (2,190)
Interest income 3,466 920 10,126 988
Total other income (expense), net 158 (1,594) 415 (1,572)
Income (loss) before taxes (43,795) (40,861) (142,677) (138,626)
Provision for income taxes 368   368  
Net loss $ (44,163) $ (40,861) $ (143,045) $ (138,626)
Net loss per share, basic (in dollars per share) $ (0.76) $ (0.79) $ (2.50) $ (2.73)
Net loss per share, diluted (in dollars per share) $ (0.76) $ (0.79) $ (2.50) $ (2.73)
Weighted-average common shares outstanding, basic (in shares) 57,874,960 51,400,922 57,154,803 50,712,452
Weighted-average common shares outstanding, diluted (in shares) 57,874,960 51,400,922 57,154,803 50,712,452
Other comprehensive loss:        
Net loss $ (44,163) $ (40,861) $ (143,045) $ (138,626)
Reclassification of losses on RareStone equity into net loss   300    
Foreign currency translation adjustment 76   49  
Unrealized gain (loss), net on marketable securities (175) 267 (70) (338)
Comprehensive loss (44,262) (40,294) (143,066) (138,964)
Product revenue        
Product revenue, net $ 22,504 $ 4,284 $ 53,194 8,094
License revenue        
Product revenue, net       $ 6,754