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Company Description (as filed with the SEC)
We are a global, late-stage, patient-focused biotechnology company engaged in the discovery and development of a diverse set of novel treatments for patients living with devastating rare and orphan diseases. We own exclusive global rights to three clinical development programs that have the potential to address significant unmet needs, each with $500 million to $1 billion estimated global market opportunities. Our lead product candidate, migalastat HCl, is an orally administered small molecule pharmacological chaperone for the treatment of Fabry disease, an LSD. The European Medicines Agency ("EMA") is currently reviewing our Marketing Authorisation Application ("MAA") for migalastat as the first potential personalized medicine for Fabry disease. ... More ...
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|Annual EPS Est:||$-1.19|
|Quarterly EPS Est:||-0.31|