Product Expertise

Gene & Cell


Our clients are those currently seeking support in navigating the landscape of advanced medicines.

Growing up in this arena, along with our partners and fellow regulators, we work to:

  • Establish benchmarks to help build robust preclinical and clinical strategies.
  • Navigate the regulatory landscape to overcome the unique considerations of rare diseases using novel technologies.
  • Assess the key advantages (and disadvantages) of accelerated approval pathways.

Unlike traditional therapies, gene therapies, as we understand them today, do not get a second chance at effectiveness. In many of our interactions with both the FDA and EMA we hear again and again that safety is one thing, but it is not the only challenge we have to overcome. While we have to ensure our therapies to be safe, we also have to delineate an effective dose.

We are helping our clients unlock value by engaging early and effectively with Regulators.

  • Early interactions to discuss target populations and development plans
  • INTERACT meetings with the Office of Tissues and Advanced Therapies
  • Early Scientific Advice meetings with the European regulators

Deliverables for Advanced Medicines

  • FDA and EMA Orphan Drug Designations (ODD)
  • Regenerative Medicines Advanced Therapy Designations (RMAT)
  • Meetings with Innovation Task Force (ITF), Committee for Advanced Therapies (CAT)
  • Scientific Advice/Protocol Assistance meetings with the EMA and National Agencies
  • OTAT INTERACT Meetings
  • Type B Meetings including pIND, EOP1, EOP2
  • Breakthrough Therapy Designation
  • Comprehensive Breakthrough Therapy Meetings (Type B)
  • Fast Track Designations
  • PRIME Designations

Given over 50% of rare diseases affect children, and focusing exclusively on orphan drug development, RareMoon’s team of senior regulatory experts have naturally provided knowledgeable and focused resources to these programs.

From nonclinical development to pivotal trials, there are distinct requirements, challenges and risks to consider in every stage of pediatric medicine development. Understanding the breadth of challenges drug manufacturers have to overcome, global agencies have put special considerations, incentives, mandates and regulations in place, as a means to aid companies in this challenging landscape. Having an intimate knowledge of the complex (and global) regulations for the development of pediatric medicines is imperative, and exactly what we have to offer.



Our deliverables unique to pediatric regulations include

  • Preparation and submission to FDA in accordance with regulatory timelines and requirements:

    • Rare Pediatric Disease Designations*
    • *Subsequent Priority Review Vouchers
  • Preparation and submission to EMA (PDCO) in accordance with regulatory timelines and requirements:

    • Early Interaction Meetings
    • Pre-submission Meetings
    • Pediatric Investigation Plans
    • EU Representation
  • Nonclinical drug development gap analysis

  • Pharmacometrics

  • Pediatric clinical study benchmark analysis

  • Strategic regulatory consulting

Drugs &


Not all drug development programs are created equal, especially in the rare disease industry.

Understanding regulatory expectations for orphan drugs is imperative when beginning your nonclinical development plans.

  • What is required to prove safety and efficacy for early nonclinical programs?
  • What if large animal models are not developed for my rare disease?
  • Are expectations for orphan clinical trials different compared to traditional drugs?

These are a just a few questions Sponsors are considering as they begin mapping out development strategies.

With our comprehensive knowledge and on-going interactions with global regulatory authorities, our experts will help you as you begin to map out your nonclinical strategy, decide when and why to apply for orphan drug designation, when to approach the agenc(ies), and what package will suffice from a scientific and regulatory perspective.

Together, we understand that a well-designed orphan drug program will maximize your chances for clinical success, and that clinical success means expedited programs and accelerated approvals.

Common Deliverables

Whether you are needing technical writing assistance or strategic program review and support, we support with:

  • IND/NDA Gap Analysis
  • IND/CTA assembly and submission (United States, Europe, Australia, Canada)
  • Authoring of Meeting Packages for EMA/FDA Interactions
  • Investigator's Brochure
  • FDA/EMA Orphan Drug Designations
  • Rare Pediatric Disease Designations
  • Fast Track Designations
  • Breakthrough Therapy Designations
  • PRIME Designations

While your message needs to be comprehensible, it also need to be brief. Our team of regulatory scientists have the experience to comprehend data and information, and author regulatory documents, IB’s, and positioning papers in a language that is effective.

A concise and focused presentation style is a challenge in preparing regulatory documents, is imperative, and allows for effective conversation with your regulators, investors and stakeholders. To achieve this, it takes smart, concise delivery backed by novel and often unconventional approaches.