FDA and EMA Orphan Drug Designations
Unmatched Expertise, Unmatched Track Record
FDA Orphan Drug Designation
Unmatched FDA Orphan Drug Designation Expertise
RareMoon is a trusted leader in obtaining FDA Orphan Drug Designations (ODDs). Our regulatory team includes former FDA orphan drug reviewers and regulatory scientists with decades of hands-on experience evaluating and submitting successful ODD applications. This insider knowledge gives RareMoon clients a measurable advantage in securing designation.
With RareMoon, sponsors receive comprehensive regulatory strategy and technical execution to support designation success.
Services Include:
- Feasibility Assessments and regulatory eligibility analysis
- Author, format, and Quality Control (QC)
- Strategic reviews of client-prepared drafts and rationales
- Guidance on FDA engagement strategy and timing
- Submission
- Post-submission support and FDA response preparation
- U.S. Resident Agent
- Annual Report preparation for orphan-designated products
EMA Orphan Designation
Proven EMA Orphan Drug Designation Success, Backed by 20+ Years of Experience with COMP
RareMoon is a recognized expert in obtaining ODDs from the European Medicines Agency for companies developing therapies for rare and serious conditions. Our regulatory team includes veteran EMA regulatory experts who have worked closely with the Committee for Orphan Medicinal Products (COMP) for over two decades, giving our clients a strategic edge in securing designation.
Our deep familiarity with EMA's evolving regulatory framework allows us to anticipate issues, guide product positioning, and craft scientifically robust justifications that meet regulatory expectations.
Services Include:
- Pre-submission strategic evaluation
- EMA-specific ODD authorship and quality review
- Pre-Submission Meeting preparation and representation
- EMA Sponsor or EU Representative support
- COMP-specific regulatory positioning and strategy
- Submission through EMA's IRIS platform
Strategic Value of FDA and EMA Orphan Drug Designation
Securing Orphan Drug Designation (ODD) early in development, whether through the FDA or the EMA, unlocks powerful regulatory and commercial advantages that can ultimately strengthen market position.
Core Regulatory Incentives
FDA
- 7 years of U.S. market exclusivity upon approval
- Waiver of Prescription Drug User Fee Act (PDUFA) fees
- Up to 25% tax credit for qualified clinical trials
EMA
- 10 years of market exclusivity in the European Union
- Reduced or waived regulatory fees, including protocol assistance
- Special support for Small and Medium-sized Enterprises (SMEs)
Additional Strategic Advantages
- Enhanced positioning for investor diligence, fundraising, and strategic partnering
- Increased visibility and credibility among patient advocacy groups and rare disease networks
- Demonstrated validation of a program's nonclinical or early clinical proof-of-concept
