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CASE STUDY | SAFETY AND EFFECTIVENESS MATTER

SAFETY AND EFFECTIVENESS MATTER

Client Success Story

the situation

The Situation

  • An ultra-rare, fatal childhood condition exists with no effective treatment
  • Condition affects 36 infants per year in the U.S.
the solution

The Solution

  • Regulatory and Quality Consulting consultants submit an Orphan Drug and Pre-IND/End of Phase 2 (EOP2) Briefing Document to FDA
  • Secure meeting with the FDA
the results

The Results

  • FDA approves small clinical trial (25 patients)
  • Orphan Designation granted
  • Improved chances of therapy development

FDA approves small clinical trial (25 patients)
Orphan Designation granted
Improved chances of therapy development

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