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Regulatory Designations

Unlocking Incentives and Accelerating Approvals.

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

For serious or life-threatening diseases that primarily affect children aged 18 years or younger.

Key Benefits:

  • Priority Review Voucher

For products addressing unmet medical needs in serious conditions.

Key Benefits:

  • Rolling review
  • More frequent meetings
  • Accelerated approval eligibility
  • Priority review if criteria met

For regenerative medicines that have preliminary clinical evidence that demonstartes 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

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

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

Provides enhanced support for innovative medicines that address unmet medical needs in the UK.

Key Benefits:

  • Reduced timelines and priority scheduling
  • Early and coordinated engagement
  • Patient-centric approaches
  • Enhanced Regulatory Support

Expedited FDA review process for drugs that address unmet medical needs or provide significant improvements in safety or effectiveness.

Key Benefits:

  • 6-month review timeline
  • Accelerated market access
  • Priority resource allocation

Incentivizes the development of high-need therapies by offering a powerful regulatory and commercial advantage to sponsors.

Key Benefits:

  • Supports mission-driven innovation
  • Voucher for accelerated FDA review
  • High market value
  • Strategic leverage
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.

Feasibility assessments

Authoring

Reviewing and finalizing applications

Submission management

US Agent / EU Sponsor Services

Agency interactions

"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
Sabrina, RareMoon's CEO

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.