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v3.26.1

Revenue, net - Narrative (Details) - USD ($) | 3 Months Ended | 6 Months Ended | 12 Months Ended |
--- | --- | --- | --- | ---
Jun. 30, 2026 | Jun. 30, 2025 | Jun. 30, 2026 | Jun. 30, 2025 | Dec. 31, 2025 | Sep. 03, 2019
Disaggregation of Revenue [Line Items] |  |  |  |  |  |
Revenue, net | $ 39,663,000 | $ 25,881,000 | $ 75,883,000 | $ 46,282,000 |  |
Contract with customer, liability | 0 |  | 0 |  | $ 0 |
Reserve or allowance for doubtful accounts | 0 |  | 0 |  | 0 |
Arimoclomol Purchase Agreement |  |  |  |  |  |
Disaggregation of Revenue [Line Items] |  |  |  |  |  |
Recognized revenue | 9,000,000.0 | 2,600,000 | 19,100,000 | 5,000,000.0 |  |
Clawback liability | 4,300,000 | 2,000,000.0 | 9,000,000.0 | 3,700,000 |  |
Estimated reserve liability |  |  | 18,600,000 |  | 15,300,000 |
MIPLYFFA |  |  |  |  |  |
Disaggregation of Revenue [Line Items] |  |  |  |  |  |
Revenue, net | 30,200,000 | 21,500,000 | 54,900,000 | 38,600,000 |  |
OLPRUVA |  |  |  |  |  |
Disaggregation of Revenue [Line Items] |  |  |  |  |  |
Revenue, net | 200,000 | 300,000 | 500,000 | 400,000 |  |
License | AZSTARYS License Agreement |  |  |  |  |  |
Disaggregation of Revenue [Line Items] |  |  |  |  |  |
Recognized revenue | 300,000 | $ 1,200,000 |  | $ 2,100,000 |  |
Contract with customer, liability, revenue recognized, including opening balance |  |  | 1,400,000 |  |  |
Contract with customer, liability | $ 0 |  | $ 0 |  | $ 0 |
License | Commave Therapeutics SA | AZSTARYS License Agreement |  |  |  |  |  |
Disaggregation of Revenue [Line Items] |  |  |  |  |  |
Milestone payments |  |  |  |  |  | $ 590,000,000.0
License | Relief Therapeutics |  |  |  |  |  |
Disaggregation of Revenue [Line Items] |  |  |  |  |  |
Royalty percentage |  |  | 10.00% |  |  |

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