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DESCRIPTION OF BUSINESS AND SIGNIFICANT ACCOUNTING POLICIES (Details 2) (USD $)
Share data in Thousands, except Per Share data, unless otherwise specified
0 Months Ended 1 Months Ended 3 Months Ended 4 Months Ended 6 Months Ended 12 Months Ended
Jun. 27, 2013
Country
Jun. 25, 2013
Jul. 31, 2013
Jun. 30, 2013
Tranche
May 31, 2012
Dec. 31, 2012
Jun. 30, 2013
Tranche
May 31, 2012
Aug. 31, 2012
Apr. 30, 2013
Age
Nov. 30, 2011
DESCRIPTION OF BUSINESS AND SIGNIFICANT ACCOUNTING POLICIES [Abstract]                      
Minimum age of children for management of nephropathic cystinosis                   6  
Period of orphan drug exclusivity for PROCYSBI   7 years                  
Number of EU member countries covered by marketing authorization for drug 28                    
Basis of Presentation [Abstract]                      
Accumulated losses       $ 175,986,000   $ 135,938,000 [1] $ 175,986,000        
Cash and cash equivalents       74,568,000 17,761,000 36,313,000 [1] 74,568,000 17,761,000     24,732,000
Net cash proceeds received from sales of common stock under ATM offering     11,200,000       23,261,000 352,000      
Inventories and Cost of Sales [Abstract]                      
Inventories       225,000   0 [1] 225,000        
Cost of sales       425,000              
Cash and Cash Equivalents [Abstract]                      
Maximum maturity period for investments considered as highly liquid investments             3 months        
Short-term Investments [Abstract]                      
Short-term investments       0   22,096,000 [1] 0        
Liabilities                      
Fair value of common stock warrants       13,500,000 19,100,000   13,500,000 19,100,000      
Fixed Assets [Abstract]                      
Useful life of significant additions and improvements in fixed assets for capitalization, minimum             1 year        
Note Payable and Debt Issuance Costs [Abstract]                      
Total amount of loan       50,000,000     50,000,000        
Number of tranches in loan       2     2        
Amount of loan per tranche       25,000,000     25,000,000        
Annual fixed interest rate (in hundredths)       10.75%     10.75%        
Income taxes [Abstract]                      
Effective tax rate (in hundredths)       0.00%   0.00% 0.00%   0.00%    
Accrued interest or penalties related to uncertain tax positions       0     0        
Unrecognized tax benefits       0     0        
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]                      
Total potentially dilutive securities (in shares)             10,549 11,283      
Net loss per share, basic and diluted (in dollars per share)       $ (0.43) $ (0.06)   $ (0.73) $ (0.35)      
Fair value assumptions used based on Black-Scholes option-pricing model [Abstract]                      
Risk-free interest rate (in hundredths)             0.92%        
Expected life             5 years        
Volatility (in hundredths)             78.00%        
Turnover rate (in hundredths)             2.50%        
Dividend rate (in hundredths)             0.00%        
Period over which current constant maturity treasury bill rates were reviewed             5 years        
Term of stock options issued             10 years        
Warrants to Purchase Common Stock [Member]
                     
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]                      
Total potentially dilutive securities (in shares)             2,213 5,188      
Options to Purchase Common Stock [Member]
                     
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]                      
Total potentially dilutive securities (in shares)             8,336 6,095      
IP license for PROCYSBI RP103 [Member]
                     
Finite-Lived Intangible Assets [Line Items]                      
Estimated useful life of intangible assets             20 years        
Out license [Member]
                     
Finite-Lived Intangible Assets [Line Items]                      
Estimated useful life of intangible assets             16 years        
Recurring [Member]
                     
Assets                      
Fair value of cash equivalents       69,142,000   35,069,000 69,142,000        
Restricted cash       213,000   163,000 213,000        
Short-term investments           22,096,000          
Total       69,355,000   57,328,000 69,355,000        
Liabilities                      
Fair value of common stock warrants       13,468,000   16,405,000 13,468,000        
Total       13,468,000   16,405,000 13,468,000        
Recurring [Member] | Level 1 [Member]
                     
Assets                      
Fair value of cash equivalents       69,142,000   35,069,000 69,142,000        
Restricted cash       0   0 0        
Short-term investments           22,096,000          
Total       69,142,000   57,165,000 69,142,000        
Liabilities                      
Fair value of common stock warrants       0   0 0        
Total       0   0 0        
Recurring [Member] | Level 2 [Member]
                     
Assets                      
Fair value of cash equivalents       0   0 0        
Restricted cash       213,000   163,000 213,000        
Short-term investments           0          
Total       213,000   163,000 213,000        
Liabilities                      
Fair value of common stock warrants       0   0 0        
Total       0   0 0        
Recurring [Member] | Level 3 [Member]
                     
Assets                      
Fair value of cash equivalents       0   0 0        
Restricted cash       0   0 0        
Short-term investments           0          
Total       0   0 0        
Liabilities                      
Fair value of common stock warrants       13,468,000   16,405,000 13,468,000        
Total       $ 13,468,000   $ 16,405,000 $ 13,468,000        
[1] Derived from the Company's audited consolidated financial statements as of December 31, 2012.