/
We are a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. We are advancing a robust and diversified pipeline of transformative medicines as part of our mission to provide access to best-in-class treatments for patients with unmet medical needs.
Margins and capital returns are both well above average: 50.09% operating margin, ROIC at 216.49%. Consistent with durable pricing power, though that alone doesn't make it a buy.
Revenue up 114.5% YoY with margins expanding 87.6pp.
Insufficient data to identify specific risks. Treat any missing metrics as a data gap, not a clean bill of health.
Profitability & Returns
Revenue (TTM)
$827M
▲ +114.5% YoY
Net Income (TTM)
-$187M
▲ +287.9% YoY
Op. Margin
-7.05%
▲ +87.6pp YoY
ROIC
-11.58%
▲ +275.9pp YoY
Cash Flow & Balance Sheet
FCF (TTM)
-$226M
▲ +715.1% YoY
Op. Cash Flow (TTM)
-$218M
▲ +760.4% YoY
Net Debt
-$1.50B
Net Cash Position
Cash & Equiv.
$1.89B
5Y CAGR: +35.4%
Continue Research