Our Vision/Mission

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