$RARE
Ultragenyx Pharmaceutical Inc.

2021 Q4 Form 10-Q Financial Statement

#000095017021002687 Filed on November 03, 2021

View on sec.gov

Income Statement

Concept 2021 Q4 2021 Q3 2020 Q4
Revenue $83.39M $81.65M $91.54M
YoY Change -8.91% 0.22% 157.19%
Cost Of Revenue $3.509M $4.175M $5.480M
YoY Change -35.97% 77.66% 7.24%
Gross Profit $79.88M $77.47M $86.06M
YoY Change -7.18% -2.08% 182.26%
Gross Profit Margin 95.79% 94.89% 94.01%
Selling, General & Admin $59.43M $53.88M $51.04M
YoY Change 16.44% 27.92% 21.87%
% of Gross Profit 74.4% 69.55% 59.31%
Research & Development $123.0M $113.4M $131.1M
YoY Change -6.17% 29.9% 57.84%
% of Gross Profit 154.0% 146.4% 152.34%
Depreciation & Amortization $3.534M $3.217M $3.130M
YoY Change 12.91% 4.11% 44.24%
% of Gross Profit 4.42% 4.15% 3.64%
Operating Expenses $182.4M $167.3M $187.6M
YoY Change -2.76% 26.95% 44.28%
Operating Profit -$102.6M -$89.83M -$101.6M
YoY Change 0.99% 78.53% 2.02%
Interest Expense -$15.66M $26.11M $80.04M
YoY Change -119.57% -356.48% 1810.26%
% of Operating Profit
Other Income/Expense, Net -$4.214M -$415.0K -$7.900M
YoY Change -46.66% -175.87% 706.12%
Pretax Income -$122.4M -$72.82M -$23.94M
YoY Change 411.44% 6.25% -73.76%
Income Tax $20.00K $182.0K $70.00K
% Of Pretax Income
Net Earnings -$122.5M -$73.00M -$24.01M
YoY Change 410.01% 6.03% -74.4%
Net Earnings / Revenue -146.85% -89.41% -26.23%
Basic Earnings Per Share -$1.08 -$0.37
Diluted Earnings Per Share -$1.786M -$1.075M -$0.37
COMMON SHARES
Basic Shares Outstanding 68.16M 67.88M
Diluted Shares Outstanding 67.88M

Balance Sheet

Concept 2021 Q4 2021 Q3 2020 Q4
SHORT-TERM ASSETS
Cash & Short-Term Investments $740.2M $737.1M $1.202B
YoY Change -38.4% -3.71% 59.09%
Cash & Equivalents $307.6M $197.5M $713.5M
Short-Term Investments $432.6M $539.6M $488.0M
Other Short-Term Assets $71.75M $69.19M $57.60M
YoY Change 24.56% 7.78% 12.06%
Inventory $16.23M $15.19M $13.05M
Prepaid Expenses
Receivables $28.43M $25.38M $23.09M
Other Receivables $0.00 $0.00 $0.00
Total Short-Term Assets $856.6M $846.9M $1.295B
YoY Change -33.87% -2.88% 52.21%
LONG-TERM ASSETS
Property, Plant & Equipment $141.2M $119.1M $73.52M
YoY Change 92.13% 37.81% 65.77%
Goodwill $44.41M $44.41M $44.41M
YoY Change 0.0% 0.0%
Intangibles $130.8M $130.9M $131.1M
YoY Change -0.25% 1.64%
Long-Term Investments $293.9M $290.5M $165.9M
YoY Change 77.13% 124.64% 404.26%
Other Assets $20.56M $16.42M $8.812M
YoY Change 133.3% 222.45% 153.87%
Total Long-Term Assets $665.8M $637.9M $464.3M
YoY Change 43.41% 60.93% 63.19%
TOTAL ASSETS
Total Short-Term Assets $856.6M $846.9M $1.295B
Total Long-Term Assets $665.8M $637.9M $464.3M
Total Assets $1.522B $1.485B $1.760B
YoY Change -13.48% 17.06% 54.96%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $17.14M $15.58M $12.92M
YoY Change 32.62% 43.55% 0.4%
Accrued Expenses $145.6M $109.7M $108.5M
YoY Change 34.16% 37.67% 30.41%
Deferred Revenue $9.076M $66.57M
YoY Change -86.37%
Short-Term Debt $0.00 $0.00 $0.00
YoY Change
Long-Term Debt Due $568.0K $500.0K
YoY Change 13.6%
Total Short-Term Liabilities $181.4M $135.9M $189.6M
YoY Change -4.35% -32.44% 83.55%
LONG-TERM LIABILITIES
Long-Term Debt $410.0K $0.00 $700.0K
YoY Change -41.43%
Other Long-Term Liabilities $1.005M $471.0K $7.349M
YoY Change -86.32% -99.87% -97.87%
Total Long-Term Liabilities $1.005M $471.0K $7.349M
YoY Change -86.32% -99.87% -97.87%
TOTAL LIABILITIES
Total Short-Term Liabilities $181.4M $135.9M $189.6M
Total Long-Term Liabilities $1.005M $471.0K $7.349M
Total Liabilities $599.8M $551.1M $605.2M
YoY Change -0.88% -8.48% 25.63%
SHAREHOLDERS EQUITY
Retained Earnings -$2.074B -$1.951B -$1.620B
YoY Change 28.03% 13.02%
Common Stock $69.00K $68.00K $67.00K
YoY Change 2.99% 15.52%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity $922.6M $933.7M $1.154B
YoY Change
Total Liabilities & Shareholders Equity $1.522B $1.485B $1.760B
YoY Change -13.48% 17.06% 54.96%

Cashflow Statement

Concept 2021 Q4 2021 Q3 2020 Q4
OPERATING ACTIVITIES
Net Income -$122.5M -$73.00M -$24.01M
YoY Change 410.01% 6.03% -74.4%
Depreciation, Depletion And Amortization $3.534M $3.217M $3.130M
YoY Change 12.91% 4.11% 44.24%
Cash From Operating Activities -$54.34M -$59.65M -$62.45M
YoY Change -12.98% -3.75% -13.31%
INVESTING ACTIVITIES
Capital Expenditures -$16.05M -$20.56M -$19.68M
YoY Change -18.46% 239.83% 71.13%
Acquisitions
YoY Change
Other Investing Activities $85.10M -$20.20M $117.6M
YoY Change -27.61% -77.48% 49.28%
Cash From Investing Activities $69.06M -$40.76M $97.90M
YoY Change -29.46% -57.44% 45.53%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 85.73M 7.550M 489.7M
YoY Change -82.49% -60.53% 55.24%
NET CHANGE
Cash From Operating Activities -54.34M -59.65M -62.45M
Cash From Investing Activities 69.06M -40.76M 97.90M
Cash From Financing Activities 85.73M 7.550M 489.7M
Net Change In Cash 100.4M -92.86M 525.2M
YoY Change -80.87% -33.0% 69.04%
FREE CASH FLOW
Cash From Operating Activities -$54.34M -$59.65M -$62.45M
Capital Expenditures -$16.05M -$20.56M -$19.68M
Free Cash Flow -$38.30M -$39.09M -$42.77M
YoY Change -10.46% -30.1% -29.35%

Facts In Submission

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<p style="text-indent:-4.749%;padding-left:4.533%;font-size:10.0pt;margin-top:0.0pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Times New Roman;">1. Organization</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Times New Roman;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">Ultragenyx Pharmaceutical Inc. (the Company) is a biopharmaceutical company incorporated in Delaware.</span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">The Company is focused on the identification, acquisition, development, and commercialization of novel products for the treatment of serious rare and ultra-rare genetic diseases. The Company operates as </span><span style="font-size:10.0pt;font-family:Times New Roman;background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">one</span><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;"> reportable segment. The Company has three commercially approved products. Crysvita® (burosumab) is approved in the United States by the U.S. Food and Drug Administration (FDA) and in Canada for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older, and is approved in the European Union (EU) and the United Kingdom, for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, adolescents, and adults. In Brazil, Colombia, and Mexico, Crysvita is approved for treatment of XLH in adult and pediatric patients one year of age and older. Crysvita is also approved in the United States by the FDA for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO), associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">The Company has also received FDA approval for Mepsevii</span><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:12.0pt;font-family:Times New Roman;">®</span><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;"> (vestronidase alfa), the first medicine approved for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. In the European Union and the United Kingdom, Mepsevii is approved under exceptional circumstances for patients of all ages for the treatment of non-neurological manifestations of MPS VII. In Brazil, Mepsevii is approved for the treatment of MPS VII for patients of all ages.</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">Dojolvi</span><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:12.0pt;font-family:Times New Roman;">®</span><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">, formerly known as UX007, is approved in the United States, Canada, and Brazil for the treatment of pediatric and adult patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD).</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">In addition to the approved products, the Company has the following ongoing clinical development programs:</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;"> </span></p><div style="margin-left:3.333%;display:flex;margin-top:4.5pt;line-height:1.3;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:0.25in;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Symbol;display:inline-block;font-size:10.0pt;font-family:Symbol;justify-content:flex-start;min-width:0.25in;"></span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">DTX401 is an adeno-associated virus 8 (AAV8) gene therapy product candidate for the treatment of patients with glycogen storage disease type Ia (GSDIa);</span></div></div><div style="margin-left:3.333%;display:flex;margin-top:4.5pt;line-height:1.3;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:0.25in;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Symbol;display:inline-block;font-size:10.0pt;font-family:Symbol;justify-content:flex-start;min-width:0.25in;"></span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with ornithine transcarbamylase (OTC) deficiency, the most common urea cycle disorder;</span></div></div><div style="margin-left:3.333%;display:flex;margin-top:4.5pt;line-height:1.3;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:0.25in;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Symbol;display:inline-block;font-size:10.0pt;font-family:Symbol;justify-content:flex-start;min-width:0.25in;"></span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">UX143 (setrusumab), which is subject to the Company's collaboration agreement with Mereo BioPharma 3 (Mereo), is a fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway and inhibits the activity of bone-forming cells for the treatment of patients with osteogenesis imperfect (OI); </span></div></div><div style="margin-left:3.333%;display:flex;margin-top:4.5pt;line-height:1.3;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:0.25in;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Symbol;display:inline-block;font-size:10.0pt;font-family:Symbol;justify-content:flex-start;min-width:0.25in;"></span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">GTX-102 is an antisense oligonucleotide (ASO), which the Company is collaborating on the development with GeneTx Biotherapeutics LLC (GeneTx) for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene;</span></div></div><div style="margin-left:3.333%;display:flex;margin-top:4.5pt;line-height:1.3;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:0.25in;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Symbol;display:inline-block;font-size:10.0pt;font-family:Symbol;justify-content:flex-start;min-width:0.25in;"></span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">UX701 is an AAV type 9 gene therapy designed to deliver stable expression of a truncated version of the ATP7B copper transporter following a single intravenous infusion to improve copper distribution and excretion from the body and reverse pathological findings of Wilson liver disease; and</span></div></div><div style="margin-left:3.333%;display:flex;margin-top:4.5pt;line-height:1.3;justify-content:flex-start;align-items:baseline;margin-bottom:0.0pt;min-width:0.25in;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Symbol;display:inline-block;font-size:10.0pt;font-family:Symbol;justify-content:flex-start;min-width:0.25in;"></span><div style="display:inline;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">UX053 is a messenger RNA (mRNA) product candidate designed for the treatment of patients with Glycogen Storage Disease Type III (GSDIII), a disease caused by a glycogen debranching enzyme (AGL) deficiency that results in glycogen accumulation in the liver and muscle. </span></div></div><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">The Company has sustained operating losses and expects such annual losses to continue over the next several years. The Company’s ultimate success depends on the outcome of its research and development and commercialization activities, for which it expects to incur additional losses in the future. Management recognizes that the Company will need to raise additional capital to fully implement its business plans. Through September 30, 2021, the Company has relied primarily on its sale of equity securities, its revenues from commercial products, its sale of future royalties, and strategic collaboration arrangements to finance its operations.</span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">The Company will likely raise additional capital through the issuance of equity, borrowings, or strategic alliances with partner companies. However, if such financing is not available at adequate levels, the Company would need to reevaluate its operating plans.</span></p>
us-gaap Use Of Estimates
UseOfEstimates
<p style="text-indent:0.0%;font-size:10.0pt;margin-top:13.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Times New Roman;">Use of Estimates</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:bold;font-size:10.0pt;font-family:Times New Roman;"> </span></p><p style="text-indent:4.533%;font-size:10.0pt;margin-top:4.5pt;line-height:1.3;font-family:Times New Roman;margin-bottom:0.0pt;text-align:left;"><span style="background-color:rgba(0,0,0,0);color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;">The accompanying Condensed Consolidated Financial Statements have been prepared in accordance with GAAP. The preparation of the Condensed Consolidated Financial Statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent liabilities and the reported amounts of expenses in the Condensed Consolidated Financial Statements and the accompanying notes. On an ongoing basis, management evaluates its estimates, including those related to clinical trial accruals, fair value of assets and liabilities, income taxes, stock-based compensation, and the liability related to the sale of future royalties. Management bases its estimates on historical experience and on various other market-specific and relevant assumptions that management believes to be reasonable under the circumstances. Actual results could differ from those estimates.</span><span style="color:rgba(0,0,0,1);white-space:pre-wrap;font-weight:normal;font-size:10.0pt;font-family:Times New Roman;"> </span></p>
CY2021Q3 us-gaap Cash And Cash Equivalents At Carrying Value
CashAndCashEquivalentsAtCarryingValue
197538000
CY2020Q3 us-gaap Cash And Cash Equivalents At Carrying Value
CashAndCashEquivalentsAtCarryingValue
197486000
CY2021Q3 us-gaap Restricted Cash Current
RestrictedCashCurrent
10000000
CY2020Q3 us-gaap Restricted Cash Current
RestrictedCashCurrent
847000
CY2021Q3 us-gaap Restricted Cash Noncurrent
RestrictedCashNoncurrent
2001000
CY2020Q3 us-gaap Restricted Cash Noncurrent
RestrictedCashNoncurrent
1921000
CY2021Q3 us-gaap Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations
CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations
209539000
CY2020Q3 us-gaap Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Including Disposal Group And Discontinued Operations
CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsIncludingDisposalGroupAndDiscontinuedOperations
200254000
CY2021Q3 us-gaap Investments Fair Value Disclosure
InvestmentsFairValueDisclosure
1004715000
CY2020Q4 us-gaap Investments Fair Value Disclosure
InvestmentsFairValueDisclosure
1327662000
CY2021Q3 us-gaap Available For Sale Securities Amortized Cost
AvailableForSaleSecuritiesAmortizedCost
918659000
CY2021Q3 us-gaap Available For Sale Securities Accumulated Gross Unrealized Gain Before Tax
AvailableForSaleSecuritiesAccumulatedGrossUnrealizedGainBeforeTax
96000
CY2021Q3 us-gaap Available For Sale Securities Accumulated Gross Unrealized Loss Before Tax
AvailableForSaleSecuritiesAccumulatedGrossUnrealizedLossBeforeTax
123000
CY2021Q3 us-gaap Available For Sale Securities
AvailableForSaleSecurities
918632000
CY2020Q4 us-gaap Available For Sale Securities Amortized Cost
AvailableForSaleSecuritiesAmortizedCost
1172646000
CY2020Q4 us-gaap Available For Sale Securities Accumulated Gross Unrealized Gain Before Tax
AvailableForSaleSecuritiesAccumulatedGrossUnrealizedGainBeforeTax
277000
CY2020Q4 us-gaap Available For Sale Securities Accumulated Gross Unrealized Loss Before Tax
AvailableForSaleSecuritiesAccumulatedGrossUnrealizedLossBeforeTax
17000
CY2020Q4 us-gaap Available For Sale Securities
AvailableForSaleSecurities
1172906000
us-gaap Available For Sale Securities Gross Realized Gain Loss Net
AvailableForSaleSecuritiesGrossRealizedGainLossNet
0
us-gaap Available For Sale Securities Gross Realized Gain Loss Net
AvailableForSaleSecuritiesGrossRealizedGainLossNet
0
CY2021Q3 us-gaap Available For Sale Securities Gross Realized Gain Loss Net
AvailableForSaleSecuritiesGrossRealizedGainLossNet
0
CY2020Q3 us-gaap Available For Sale Securities Gross Realized Gain Loss Net
AvailableForSaleSecuritiesGrossRealizedGainLossNet
0
CY2021Q3 us-gaap Inventory Work In Process
InventoryWorkInProcess
9546000
CY2020Q4 us-gaap Inventory Work In Process
InventoryWorkInProcess
7184000
CY2021Q3 us-gaap Inventory Finished Goods
InventoryFinishedGoods
5645000
CY2020Q4 us-gaap Inventory Finished Goods
InventoryFinishedGoods
5864000
CY2021Q3 us-gaap Inventory Net
InventoryNet
15191000
CY2020Q4 us-gaap Inventory Net
InventoryNet
13048000
CY2021Q3 rare Research Clinical Study And Manufacturing Expenses
ResearchClinicalStudyAndManufacturingExpenses
22258000
CY2020Q4 rare Research Clinical Study And Manufacturing Expenses
ResearchClinicalStudyAndManufacturingExpenses
25875000
CY2021Q3 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
55164000
CY2020Q4 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
58176000
CY2021Q3 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
32238000
CY2020Q4 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
24440000
CY2021Q3 us-gaap Accrued Liabilities Current
AccruedLiabilitiesCurrent
109660000
CY2020Q4 us-gaap Accrued Liabilities Current
AccruedLiabilitiesCurrent
108491000
CY2021Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
81647000
CY2020Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
81470000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
268017000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
179488000
CY2021Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
81647000
CY2020Q3 us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
81470000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
268017000
us-gaap Revenue From Contract With Customer Excluding Assessed Tax
RevenueFromContractWithCustomerExcludingAssessedTax
179488000
CY2020Q4 rare Contract With Customer Assets Liability Current
ContractWithCustomerAssetsLiabilityCurrent
-66568000
rare Contract With Customer Assets Liability Additions During Period
ContractWithCustomerAssetsLiabilityAdditionsDuringPeriod
-2390000
rare Contract With Customer Assets Liability Additions During Period
ContractWithCustomerAssetsLiabilityAdditionsDuringPeriod
-154621000
rare Contract With Customer Assets Liability Deductions During Period
ContractWithCustomerAssetsLiabilityDeductionsDuringPeriod
76767000
rare Contract With Customer Assets Liability Deductions During Period
ContractWithCustomerAssetsLiabilityDeductionsDuringPeriod
51723000
CY2021Q3 rare Contract With Customer Assets Liability Current
ContractWithCustomerAssetsLiabilityCurrent
7809000
CY2020Q3 rare Contract With Customer Assets Liability Current
ContractWithCustomerAssetsLiabilityCurrent
-102898000
CY2021Q3 rare Percentage Of Gross Accounts Receivable Balance
PercentageOfGrossAccountsReceivableBalance
0.57
CY2020Q4 rare Percentage Of Gross Accounts Receivable Balance
PercentageOfGrossAccountsReceivableBalance
0.71
CY2019Q4 rare Liability Related To Sale Of Future Royalties
LiabilityRelatedToSaleOfFutureRoyalties
315369000
CY2020 rare Non Cash Collaboration Royalty Revenue
NonCashCollaborationRoyaltyRevenue
12995000
CY2020 rare Non Cash Interest Expense On Liability Related To Sale Of Future Royalties
NonCashInterestExpenseOnLiabilityRelatedToSaleOfFutureRoyalties
33291000
CY2020Q4 rare Liability Related To Sale Of Future Royalties
LiabilityRelatedToSaleOfFutureRoyalties
335665000
rare Non Cash Collaboration Royalty Revenue
NonCashCollaborationRoyaltyRevenue
13210000
rare Non Cash Interest Expense On Liability Related To Sale Of Future Royalties
NonCashInterestExpenseOnLiabilityRelatedToSaleOfFutureRoyalties
25618000
CY2021Q3 rare Liability Related To Sale Of Future Royalties
LiabilityRelatedToSaleOfFutureRoyalties
348073000
CY2021Q3 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
26990000
CY2020Q3 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
20341000
us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
78430000
us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
62922000
CY2021Q3 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
8317206
CY2020Q3 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
8662701
us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
8304317
us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
8696942
CY2021Q3 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
81000
CY2020Q4 us-gaap Accumulated Other Comprehensive Income Loss Foreign Currency Translation Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossForeignCurrencyTranslationAdjustmentNetOfTax
429000
CY2021Q3 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
-27000
CY2020Q4 us-gaap Accumulated Other Comprehensive Income Loss Available For Sale Securities Adjustment Net Of Tax
AccumulatedOtherComprehensiveIncomeLossAvailableForSaleSecuritiesAdjustmentNetOfTax
260000
CY2021Q3 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
54000
CY2020Q4 us-gaap Accumulated Other Comprehensive Income Loss Net Of Tax
AccumulatedOtherComprehensiveIncomeLossNetOfTax
689000

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