XML 63 R48.htm IDEA: XBRL DOCUMENT v3.24.0.1
License and Collaboration Agreements - Additional Information (Detail)
$ in Thousands, shares in Millions
1 Months Ended 12 Months Ended
Dec. 21, 2019
USD ($)
shares
Nov. 30, 2021
USD ($)
Oct. 31, 2021
USD ($)
Jul. 31, 2021
USD ($)
Nov. 30, 2019
USD ($)
Apr. 30, 2019
USD ($)
Jul. 31, 2017
Jun. 30, 2016
USD ($)
Dec. 31, 2023
USD ($)
Milestone
shares
Dec. 31, 2022
USD ($)
Dec. 31, 2021
USD ($)
Mar. 31, 2024
USD ($)
Feb. 28, 2021
USD ($)
Dec. 31, 2019
USD ($)
Aug. 08, 2018
USD ($)
shares
Dec. 31, 2017
USD ($)
Dec. 31, 2016
USD ($)
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Issuance costs     $ 26,500                            
Product revenues, net                 $ 1,144,876 $ 843,769 $ 612,401            
Impairment of strategic investments                 30,321 2,575 4,488            
Contingent consideration                 38,100 36,900              
Intangible asset, net                 29,620 7,578              
Cost of sales (excluding amortization of in-licensed rights)                 150,343 139,989 97,049            
Amortization of in-licensed rights                 1,559 714 706            
Development milestone and settlement upfront fee recognized as research and development expense                 13,200 32,600 50,300            
ELEVIDYS [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Product revenues, net                 200,356 0              
US [Member] | ELEVIDYS [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Product revenues, net                 200,356 0              
Minimum [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Percentage of royalty payments             4.00%                    
Maximum [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Percentage of royalty payments             8.00%                    
Genethon [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Research and development expense                 $ 0 0 4,000            
Up-front cash payment under development         $ 157,500                        
Up-front cash payment under sales milestone         $ 78,800                        
Up-front cash payment under development percentage         75.00%                        
Number of development milestone | Milestone                 0                
Other Licensed Products [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Up-front cash payment under development percentage         25.00%                        
Myonexus Therapeutics, Inc. [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Final exercise price           $ 178,300                      
Additional development milestone payments to be paid           $ 200,000                      
Contingent consideration                 $ 37,700 35,500              
Lacerta Therapeutics [Member] | Series A Preferred Stock                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Impairment of strategic investments                 30,000                
BioMarin Pharmaceutical, Inc. [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Regulatory and sales milestone payments                 0                
Intangible asset, net                               $ 6,600  
Sales milestone payment recorded as an in-license right                 0                
Royalty expense                 17,600 30,400 31,400            
BioMarin Pharmaceutical, Inc. [Member] | AMONDYS 45 [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Regulatory milestone payable                         $ 10,000        
BioMarin Pharmaceutical, Inc. [Member] | Intellectual Property                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Amortization of in-licensed rights                 2,300                
BioMarin Pharmaceutical, Inc. [Member] | US [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Royalty payment expiry period   2024-03                              
BioMarin Pharmaceutical, Inc. [Member] | EU [member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Royalty payment expiry period   2024-12                              
BioMarin Pharmaceutical, Inc. [Member] | Minimum [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Percentage of royalty payments   4.00%                              
BioMarin Pharmaceutical, Inc. [Member] | Maximum [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Regulatory and sales milestone payments   $ 50,000                              
Percentage of royalty payments   5.00%                              
BioMarin Pharmaceutical, Inc. [Member] | Maximum [Member] | Other Countries [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Royalty payment expiry period   2024-12                              
University of Western Australia [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Regulatory and sales milestone payments                 0                
Sales milestone payment recorded as an in-license right                 0                
Royalty expense                 11,800 10,500 7,700            
Carrying value of intangible assets                 700                
University of Western Australia [Member] | EXONDYS 51 [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Sales milestone payment recorded as an in-license right                                 $ 1,000
University of Western Australia [Member] | VYONDYS 53 [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Sales milestone payment recorded as an in-license right                           $ 500      
University of Western Australia [Member] | AMONDYS 45 [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Sales milestone payment recorded as an in-license right                         $ 500        
University of Western Australia [Member] | Maximum [Member] | EXONDYS 51 [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Payments to UWA on successful achievement of certain development and regulatory milestones               $ 26,000                  
Collaborative Arrangement                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Payment for option exercise                 165,800   0            
Contingent research milestone payments                 5,100 6,000 3,000            
Collaborative Arrangement | Research and Development Expense [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Up-front and development milestone expenses                 7,500 8,500              
Collaborative Arrangement | ELEVIDYS [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Collaboration and other receivable                 7,100                
Collaborative Arrangement | Forecast [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Collaboration and other receivable                       $ 48,000          
Collaborative Arrangement | Maximum [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Development milestone and settlement upfront fee recognized as research and development expense                 3,200                
Contingent research milestone payments                 $ 34,800                
Collaborative Arrangement | Roche Holding A.G. [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Proceeds from license and collaborative agreement $ 1,200,000                                
Issuance costs $ 312,100                                
Stock issued during period shares collaboration and license agreement | shares 2.5                                
Payment for option exercise $ 485,000                                
Single combined performance obligation 348,700                                
Stock options exercised or expired | shares                 0.0                
Product revenues, net                 $ 98,500 89,200 89,500            
Deferred revenue                 487,400                
Deferred revenue, current                 50,400                
Deferred revenue separate material options right                 485,000 485,000              
Research and development expense                 106,900 117,800 90,500            
Collaboration receivable                 29,800 41,800              
Collaborative Arrangement | Roche Holding A.G. [Member] | ELEVIDYS [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Collaboration and other receivable                 9,200 0              
Inventory costs                 1,800                
Collaborative Arrangement | Roche Holding A.G. [Member] | Maximum [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Regulatory and sales milestones payment received $ 1,700,000                                
Collaborative Arrangement | Genethon [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Research and development expense                 6,600 $ 3,500 $ 11,700            
Up-front cash payment under development         $ 28,000                        
Series A Preferred Stock Purchase Agreement | Lacerta Therapeutics [Member] | Series A Preferred Stock                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Up-front payment | shares                             4.5    
Series A Preferred Stock Purchase Agreement | Lacerta Therapeutics [Member] | Other Noncurrent Assets [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Deferred revenue                             $ 30,000    
License And Purchase Agreement | Lacerta Therapeutics [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Deferred revenue                             $ 38,000    
Nationwide License Agreement [Member] | ELEVIDYS [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Carrying value of intangible assets                 22,200                
Nationwide License Agreement [Member] | Nationwide Children's Hospital [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Sublicense payment       $ 38,000                          
Cost of sales (excluding amortization of in-licensed rights)                 6,000                
Carrying value of intangible assets                 23,000                
Nationwide License Agreement [Member] | Nationwide Children's Hospital [Member] | ELEVIDYS [Member]                                  
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items]                                  
Carrying value of intangible assets                 $ 10,000