Our Vision is to be recognized as a leading global orphan drug company committed to restoring health and transforming the lives of people with rare, debilitating, and often life threatening conditions.
Our Mission is to develop novel, best-in-class drugs that optimize health outcomes and improve quality of life for patients with rare diseases. Our strategy to achieve this mission is to:
- Advance development of our current product candidates
- Commercialize our products in the United States
- Accelerate development and maximize potential of our product candidates outside the United States through strategic alliances and collaborations with other pharmaceutical companies
- Expand our current portfolio by leveraging our expertise in development and commercialization to identify and in-license additional orphan drug candidates with significant commercial potential