Regulatory Designations
Unlocking Incentives and Accelerating Approvals.
FDA and EMA Orphan Drug Designation (ODD)
+
Incentivizes development of treatments for rare diseases affecting fewer than 200,000 people in the US or less than 5 in 10,000 in the EU.
Key Benefits:
- 7 years market exclusivity (FDA)
- 10 years market exclusivity (EMA)
- Tax credits (FDA)
- Protocol assistance (EMA)
- Reduced fees
FDA Rare Pediatric Disease Designation (RPD)
+
For serious or life-threatening diseases that primarily affect children aged 18 years or younger.
Key Benefits:
- Priority Review Voucher
Designation Action Plan
+
This deliverable includes a comprehensive FDA Designation Action Plan, a structured, forward-looking assessment to identify, qualify, and leverage FDA acceleration mechanisms.
Key Benefits:
- Evaluation of program across all FDA designations and pathways
- Feasibility assessment
- Identify opportunities to stack and sequence designations
- Deliver a clear timeline and action plan
FDA Regenerative Medicine Advanced Therapy Designation (RMAT)
+
For regenerative medicines that have preliminary clinical evidence that demonstrates the potential to address an unmet need in a serious or life-threatening condition.
Key Benefits:
- Intensive FDA guidance
- Rolling review
- Priority review
- Accelerated approval eligibility
FDA Breakthrough Therapy Designation
+
For drugs that treat serious conditions and have preliminary clinical evidence of substantial improvement over existing treatments.
Key Benefits:
- Intensive FDA guidance
- Rolling review
- Priority review
- Accelerated approval eligibility
EMA Priority Medicines (PRIME)
+
Supports development of medicines addressing unmet medical needs through enhanced scientific advice and regulatory support.
Key Benefits:
- Enhanced scientific advice
- Early dialogue
- Accelerated assessment
- Dedicated contact point
Rare Disease Pilot Programs
+
The FDA has implemented several programs that aim to foster collaboration and provide a flexible framework to rare disease drug developers.
Key Benefits:
- START (Support for Clinical Trials Advancing Rare Disease Therapeutics)
- RDEA (Rare Disease Endpoint Advancement)
- RDEP (Rare Disease Evidence Principles)
FDA Priority Review
+
Expedited FDA review process that shortens the review timeline from the standard 10 months to 6 months for drugs that offer significant improvements in safety or effectiveness.
Key Benefits:
- 6-month review timeline
- Accelerated market access
- Available through Priority Review Voucher programs
- Strategic leverage for rare disease sponsors
250+
FDA and EMA ORPHAN DRUG DESIGNATIONS
100+
RARE PEDIATRIC DISEASE DESIGNATIONS
40+
FAST TRACK, BREAKTHROUGH THERAPY, RMAT DESIGNATIONS
400+
UNIQUE ASSETS SUPPORTED
(Last Updated June 2025)
Our Process in Securing Regulatory Designations
Regulatory designations like RMAT, Breakthrough Therapy, Accelerated Approval, and PRIME can de-risk development, unlock funding, and provide earlier access to regulators and markets. At RareMoon, we specialize in securing these strategic designations. With hundreds of successful submissions and a high first-time success rate, our team knows how to navigate nuanced criteria, craft strong justifications, and position programs for success, before and after designation is granted.
From eligibility assessment through post-designation planning, we bring clarity, momentum, and confidence to every step. Our proven, internally developed process ensures programs are strategically positioned for success and data readiness when approaching regulators.
“Beyond securing your designation, our real value lies in helping you understand when to apply, how to position your data, and how to align your messaging for success.”
~Sabrina, RareMoon’s CEO
Feasibility assessments
Authoring
Reviewing and finalizing applications
Submission management
US Agent / EU Sponsor Services
Agency interactions
What You Get With RareMoon
High Success Rates
With streamlined processes and deep expertise, we expedite the process of securing ODD and crafting well-aligned applications that meet regulatory expectations.
Accelerated Timelines
With a team of highly skilled regulatory writers, we know exactly where to begin and how to navigate the process efficiently, ensuring swift and accurate document preparation from start to finish.
Expert Involvement
Our team of experts is ready to support, with MS, PhD, and MD-level professionals directly involved in your projects.
End-to-End Service
From initial feasibility assessments through to post-submission follow-up.
Is your data designation-ready?
Schedule a consultation to explore designation opportunities.
Related Services
Orphan Drug Designations
Expert guidance for FDA and EMA orphan drug designation applications.
Learn moreBreakthrough Therapy Designation
Navigate FDA Breakthrough Therapy Designation with expert strategic guidance.
Learn moreRMAT Designation
FDA Regenerative Medicine Advanced Therapy designation for cell and gene therapies.
Learn moreRare Pediatric Disease Designation
Strategic support for FDA Rare Pediatric Disease Designation and Priority Review Vouchers.
Learn moreRelated Insights
Navigating FDA's Rare Disease Programs: A Practical Framework for Sponsors
A practical overview of the FDA rare disease and advanced therapy landscape in early 2026, with guidance on how to layer designations, pilots, and pathways to support program execution.
Read: Navigating FDA's Rare Disease Programs: A Practical Framework for SponsorsAccelerating Development in an Evolving FDA Landscape
With the FDA landscape shifting faster than ever, RareMoon is helping sponsors identify and leverage opportunities through our FDA Designation Action Plan, a structured approach to anticipate, prioritize, and pursue various FDA acceleration mechanisms.
Read: Accelerating Development in an Evolving FDA Landscape