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Revenue, net - Narrative (Details) - USD ($)	3 Months Ended		6 Months Ended		12 Months Ended
Revenue, net - Narrative (Details) - USD ($)	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%