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Delivering Protein Therapeutics to the CNS

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
    • Dramatically smaller clinical trials
    • <100 patients for gaucher disease phase iii clinical trials
    • Tax credits
    • Phase III dramatically 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