Segment Information
Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the CODM or decision-making group in making decisions on how to allocate resources and assess performance. Our CODM is our CEO. Our CEO views our operations and manages our business as one operating segment, which derives its revenues from the development and commercialization of therapies for patients with rare diseases.
Our CEO manages and allocates resources to the operations of our company on a total company basis by assessing the overall level of resources available and how to best deploy these resources across functions and research and development projects that are in line with our long-term company-wide strategic goals. In making these decisions, our CEO uses consolidated financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets. The CODM performs this assessment based on our consolidated net (loss) income. Through this analysis, the CODM assesses performance by comparing actual consolidated net (loss) income versus the budget, and then decides how to allocate resources to invest in our research and development programs. The measure of segment assets is reported on the consolidated balance sheets as total assets.
The following table contains additional information on our consolidated revenue and net (loss) income, including significant segment expenses:
(In thousands) Years Ended December 31:202520242023
Product revenue, net - U.S.$49,170 $36,395 $26,823 
Product revenue, net - Rest of world4,858 103 — 
PK activator (PYRUKYND®/AQVESME™) direct expenses - research and development(108,087)(112,720)(101,322)
Compensation and related expenses - research and development(121,645)(114,618)(108,484)
Total selling, general and administrative expenses(180,280)(156,784)(119,903)
Gain on sale of contingent payments— 889,136 — 
Milestone payment from gain on sale of oncology business— 200,000 — 
Other segment items*(56,797)(67,787)(49,202)
Net (loss) income$(412,781)$673,725 $(352,088)
*Other segment items primarily include cost of sales, other research and development expenses, interest income and income taxes.

Historical Timeline

Fiscal YearFiled
2025Feb 12, 2026Showing above
2024Feb 13, 2025

About Segments Disclosures

Segment disclosures break a company into its reportable operating units, revealing revenue, profit, and asset allocation that consolidated financial statements obscure. Under ASC 280, segments must match how the chief operating decision maker views the business, providing a window into internal management structure and resource allocation priorities.

Key signals: compare segment margins to identify which units drive profitability and which destroy value. Watch for changes in the number of reportable segments — segment aggregation or disaggregation often coincides with strategic shifts or attempts to obscure declining performance. Intersegment elimination patterns reveal internal pricing practices. The reconciliation between segment totals and consolidated figures exposes corporate overhead allocation and unallocated items. Geographic revenue concentration highlights regulatory and currency exposure. Compare segment-level capital expenditure against segment revenue to assess where management is investing for future growth versus harvesting existing assets.