Pay vs Performance Disclosure - USD ($) | 12 Months Ended | 36 Months Ended |
Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2022 |
Pay vs Performance Disclosure [Table] | | | | | |
Pay vs Performance [Table Text Block] | | | | | Pay versus Performance As required by Section 953(a) of the Dodd-Frank Wall Street Reform and Consumer Protection Act, and Item 402(v) of Regulation S-K, we are providing the following information about the relationship between Compensation Actually Paid (CAP) and certain financial performance of the Company. For further information concerning the Company’s pay for performance philosophy and how the Company’s aligns executive compensation with the Company’s performance, refer to “Executive Compensation – Compensation Discussion and Analysis.” Average Value of Initial Fixed $100 Year Summary (1) Compensation (2) Summary (3) Average (4) Total (5) Peer Group (6) Net Income (7) Revenue (8) 2022 $ 12,140,330 $ 3,311,432 $ 3,911,773 $ (175,590) $ 108 $ 111 $ (707,421) $ 363,329 2021 $ 9,522,738 $ (2,152,804) $ 3,314,256 $ (1,211,638) $ 197 $ 125 $ (454,025) $ 351,406 2020 $ 5,144,220 $ 22,726,743 $ 2,706,102 $ 8,015,000 $ 324 $ 126 $ (186,566) $ 271,030 (1) The dollar amounts reported are the amounts in the “Total” column of the Summary Compensation Table in each applicable year for Dr. Kakkis, who served as our Chief Executive Officer during 2020, 2021 and 2022. (2) The dollar amounts reported represent the amount of CAP, as computed in accordance with SEC rules. The dollar amounts do not reflect the actual amount of compensation earned by or paid during the applicable year. In accordance with SEC rules, the following adjustments were made to total compensation to determine the CAP: Compensation Actually Paid to PEO 2022 2021 2020 Summary Compensation Table Total $ 12,140,330 $ 9,522,738 $ 5,144,220 Less, (10,773,976) (8,246,092) (3,709,589) Plus, (6,264,762) 4,152,033 10,163,438 Plus (2,934,552) (5,753,632) 9,346,926 Plus (less), (1,385,132) (1,827,851) 1,781,748 Less, — — — Compensation Actually Paid to Dr. Kakkis $ 3,311,432 $ (2,152,804) $ 22,726,743 (3) The dollar amounts reported represent the average of the amounts reported for the Company’s named executive officers (NEOs) as a group (excluding our CEO) in the “Total” column of the Summary Compensation Table in each applicable year. The names of each of the NEOs (excluding our CEO) included for purposes of calculating the average amounts in each applicable year are as follows: (i) for 2022 and 2021, Dr. Bedrosian, Mr. Harris, Mr. Pinion and Ms. Dier; and (ii) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp. (4) The dollar amounts reported represent the average amount of CAP to the NEOs as a group (excluding our CEO), as computed in accordance with SEC rules. The dollar amounts do not reflect the actual average amount of compensation earned by or paid to the NEOs as a group (excluding our CEO) during the applicable year. In accordance with the SEC rules, the following adjustments were made to average total compensation for the NEOs as a group (excluding our CEO) for each year to determine the compensation actually paid, using the same methodology described above in Note 2: Compensation Actually Paid to Non-PEO NEOs 2022 2021 2020 Summary Compensation Table Total $ 3,911,773 $ 3,314,256 $ 2,706,102 Less, (3,130,468) (2,519,639) (2,027,088) Plus, (a) 1,264,910 1,268,695 4,150,071 Plus (less), (b) (784,234) (2,359,539) 2,748,610 Plus (less), (c) (573,020) (915,411) 437,305 Less, (d) (864,551) — — Compensation Actually Paid to Non-PEO NEOs $ (175,590) $ (1,211,638) $ 8,015,000 (5) Cumulative TSR is calculated by dividing (a) the sum of (i) the cumulative amount of dividends for the measurement period, assuming dividend reinvestment, and (ii) the difference between the Company’s share price at the end and the beginning of the measurement period by (b) the Company’s share price at the beginning of the measurement period. The beginning of the measurement period for each year in the table is December 31, 2019. (6) Represents the weighted peer group TSR, weighted according to the respective companies’ stock market capitalization at the beginning of each period for which a return is indicated. The peer group used for this purpose is the following published industry index: Nasdaq Biotechnology Index. (7) The dollar amounts reported represent the amount of net income calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year. (8) The dollar amounts reported represent the amount of revenue calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year. |
Company Selected Measure Name | [1] | | | | Revenue |
Named Executive Officers, Footnote [Text Block] | | | | | (3)The dollar amounts reported represent the average of the amounts reported for the Company’s named executive officers (NEOs) as a group (excluding our CEO) in the “Total” column of the Summary Compensation Table in each applicable year. The names of each of the NEOs (excluding our CEO) included for purposes of calculating the average amounts in each applicable year are as follows: (i) for 2022 and 2021, Dr. Bedrosian, Mr. Harris, Mr. Pinion and Ms. Dier; and (ii) for 2020, Dr. Bedrosian, Ms. Dier, Mr. Harris, Mr. Pinion, and Shalini Sharp. |
Peer Group Issuers, Footnote [Text Block] | | | | | (6)Represents the weighted peer group TSR, weighted according to the respective companies’ stock market capitalization at the beginning of each period for which a return is indicated. The peer group used for this purpose is the following published industry index: Nasdaq Biotechnology Index. |
PEO Total Compensation Amount | [2] | $ 12,140,330 | $ 9,522,738 | $ 5,144,220 | |
PEO Actually Paid Compensation Amount | [3] | 3,311,432 | (2,152,804) | 22,726,743 | |
Adjustment To PEO Compensation, Footnote [Text Block] | | | | | (2)The dollar amounts reported represent the amount of CAP, as computed in accordance with SEC rules. The dollar amounts do not reflect the actual amount of compensation earned by or paid during the applicable year. In accordance with SEC rules, the following adjustments were made to total compensation to determine the CAP: Compensation Actually Paid to PEO 2022 2021 2020 Summary Compensation Table Total $ 12,140,330 $ 9,522,738 $ 5,144,220 Less, (10,773,976) (8,246,092) (3,709,589) Plus, (6,264,762) 4,152,033 10,163,438 Plus (2,934,552) (5,753,632) 9,346,926 Plus (less), (1,385,132) (1,827,851) 1,781,748 Less, — — — Compensation Actually Paid to Dr. Kakkis $ 3,311,432 $ (2,152,804) $ 22,726,743 |
Non-PEO NEO Average Total Compensation Amount | [4] | 3,911,773 | 3,314,256 | 2,706,102 | |
Non-PEO NEO Average Compensation Actually Paid Amount | [5] | $ (175,590) | (1,211,638) | 8,015,000 | |
Adjustment to Non-PEO NEO Compensation Footnote [Text Block] | | | | | (4)The dollar amounts reported represent the average amount of CAP to the NEOs as a group (excluding our CEO), as computed in accordance with SEC rules. The dollar amounts do not reflect the actual average amount of compensation earned by or paid to the NEOs as a group (excluding our CEO) during the applicable year. In accordance with the SEC rules, the following adjustments were made to average total compensation for the NEOs as a group (excluding our CEO) for each year to determine the compensation actually paid, using the same methodology described above in Note 2: Compensation Actually Paid to Non-PEO NEOs 2022 2021 2020 Summary Compensation Table Total $ 3,911,773 $ 3,314,256 $ 2,706,102 Less, (3,130,468) (2,519,639) (2,027,088) Plus, (a) 1,264,910 1,268,695 4,150,071 Plus (less), (b) (784,234) (2,359,539) 2,748,610 Plus (less), (c) (573,020) (915,411) 437,305 Less, (d) (864,551) — — Compensation Actually Paid to Non-PEO NEOs $ (175,590) $ (1,211,638) $ 8,015,000 |
Compensation Actually Paid vs. Net Income [Text Block] | | | | | |
Compensation Actually Paid vs. Company Selected Measure [Text Block] | | | | | |
Tabular List [Table Text Block] | | Performance Measures As described in greater detail in “Executive Compensation – Compensation Discussion and Analysis,” the Company’s executive compensation program reflects a variable pay-for-performance philosophy. The metrics that the Company uses for both our long-term and short-term incentive awards are selected based on an objective of incentivizing our NEOs to increase the value of our enterprise for our shareholders. The most important performance measures used by the Company to link executive compensation actually paid to the Company’s NEOs, for the most recently completed fiscal year, to the Company’s performance are as follows: • Total Revenue • Relative Total Stockholder Return • Net Cash Used in Operations • Clinical Development of Product Candidates | | | |
Total Shareholder Return Amount | [6] | $ 108 | 197 | 324 | |
Peer Group Total Shareholder Return Amount | [7] | 111 | 125 | 126 | |
Net Income (Loss) | [8] | $ (707,421,000) | $ (454,025,000) | $ (186,566,000) | |
Company Selected Measure Amount | [1] | 363,329,000 | 351,406,000 | 271,030,000 | |
PEO Name | | | | | Dr. Kakkis |
Additional 402(v) Disclosure [Text Block] | | | | | |
Measure [Axis]: 1 | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Measure Name | | Total Revenue | | | |
Measure [Axis]: 2 | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Measure Name | | Relative Total Stockholder Return | | | |
Measure [Axis]: 3 | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Measure Name | | Net Cash Used in Operations | | | |
Measure [Axis]: 4 | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Measure Name | | Clinical Development of Product Candidates | | | |
PEO [Member] | Less, value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | $ (10,773,976) | $ (8,246,092) | $ (3,709,589) | |
PEO [Member] | Plus, year end fair value of outstanding and unvested equity awards granted during the year [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | (6,264,762) | 4,152,033 | 10,163,438 | |
PEO [Member] | Plus (less), year over year change in fair value of outstanding and unvested equity awards granted in prior years [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | (2,934,552) | (5,753,632) | 9,346,926 | |
PEO [Member] | Plus (less), year over year change in fair value of equity awards granted in prior years that vested in the year [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | (1,385,132) | (1,827,851) | 1,781,748 | |
Non-PEO NEO [Member] | Less, value of “Stock Awards” and “Option Awards” reported in Summary Compensation Table [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | (3,130,468) | (2,519,639) | (2,027,088) | |
Non-PEO NEO [Member] | Plus, year end fair value of outstanding and unvested equity awards granted during the year [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | 1,264,910 | 1,268,695 | 4,150,071 | |
Non-PEO NEO [Member] | Plus (less), year over year change in fair value of outstanding and unvested equity awards granted in prior years [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | (784,234) | (2,359,539) | 2,748,610 | |
Non-PEO NEO [Member] | Plus (less), year over year change in fair value of equity awards granted in prior years that vested in the year [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | (573,020) | $ (915,411) | $ 437,305 | |
Non-PEO NEO [Member] | Less, prior year-end fair value of any equity awards that failed to meet vesting conditions in the year [Member] | | | | | |
Pay vs Performance Disclosure [Table] | | | | | |
Adjustment to Compensation Amount | | $ (864,551) | | | |
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[1](8)The dollar amounts reported represent the amount of revenue calculated in accordance with GAAP reflected in the Company’s audited financial statements for the applicable year.[2](1)The dollar amounts reported are the amounts in the “Total” column of the Summary Compensation Table in each applicable year for Dr. Kakkis, who served as our Chief Executive Officer during 2020, 2021 and 2022.[3](2)The dollar amounts reported represent the amount of CAP, as computed in accordance with SEC rules. The dollar amounts do not reflect the actual amount of compensation earned by or paid during the applicable year. In accordance with SEC rules, the following adjustments were made to total compensation to determine the CAP: Compensation Actually Paid to PEO 2022 2021 2020 Summary Compensation Table Total $ 12,140,330 $ 9,522,738 $ 5,144,220 Less, (10,773,976) (8,246,092) (3,709,589) Plus, (6,264,762) 4,152,033 10,163,438 Plus (2,934,552) (5,753,632) 9,346,926 Plus (less), (1,385,132) (1,827,851) 1,781,748 Less, — — — Compensation Actually Paid to Dr. Kakkis $ 3,311,432 $ (2,152,804) $ 22,726,743 Compensation Actually Paid to Non-PEO NEOs 2022 2021 2020 Summary Compensation Table Total $ 3,911,773 $ 3,314,256 $ 2,706,102 Less, (3,130,468) (2,519,639) (2,027,088) Plus, (a) 1,264,910 1,268,695 4,150,071 Plus (less), (b) (784,234) (2,359,539) 2,748,610 Plus (less), (c) (573,020) (915,411) 437,305 Less, (d) (864,551) — — Compensation Actually Paid to Non-PEO NEOs $ (175,590) $ (1,211,638) $ 8,015,000 | |