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INNOVATIVE SCIENCE

Delivering Protein Therapeutics to the CNS for Gaucher Disease

Orphan drugs are often brought to market quickly, through addressing small, targeted, often ignored patient populations, thus increasing the potential for strategic partnerships  with Top Tier Pharmaceutical Companies

Key Advantages:

  • Shorter timeline to market
  • Requires significantly less resources
    • <100 patients for Gaucher Disease Phase III clinical trials
    • <550 patients for all orphan drug Phase II/III studies
    • Tax credits (<50% of R&D costs & clinical trial incentives)
    • Phase III up to 50-75% cheaper than non-orphan drugs
    • Waived FDA fees and protocol assistance
  • FDA is often more flexible with approvals, resulting in shorter timeline to market
  • Marketing exclusivity (7 years from approval in the U.S.)
  • Lower marketing costs as patients are easily located
  • Faster uptake and market acceptance
  • Generally well reimbursed