A microbiome describes the collection of microbial life (microbiota) that inhabits a specific environment and includes microorganisms as diverse as bacteria, yeasts, fungi, archaea and viruses such as phages. In humans, these organisms are resident primarily in the gastrointestinal tract but are also found on the skin, genitals and in the lungs. Although the extent of the relationship between the microbiome and human health is not fully known yet. The scientific community is gaining a better understanding of the role of these microorganisms in the development of pathologies such as obesity, gastrointestinal diseases (typically, Irritable Bowel Syndrome (IBS) or Crohn’s disease) depression and allergies. It is now possible to approach these and other challenges from a new angle. This includes the development of new foodstuffs to maintain beneficial flora, to restore a flora in dysbiosis as well as the development of new drugs that offer more targeted possibilities.
Microbiome products are typically designated as prebiotics, probiotics or postbiotics, or a combination thereof (Symbiotics). However, these are not regulatory product categories. Applicable status may vary depending on the product intended purpose (e.g. target indication and population claims), composition, route of administration and mode of action. They can be either drug, Live Biotherapeutic Products LBPs, biological medicinal products, functional foods, food/dietary supplements, medical devices, transplants or tissues & cells. Some are specific to microbiome products (e.g. Fecal Microbiota Transplant (FMT)) while others also apply to other types of health products.
Key challenges encompass:
- Managing the presence of live microorganisms in production sites
- Controlling the risk of transfer of antibiotic resistance between the live microorganism(s) given to the patient and its microflora
- Ensuring and controlling the viability of a LBP to its site of action
- Determining whether conventional nonclinical pharmacokinetics (e.g., ADME) and toxicological studies are applicable
- Defining appropriate endpoints for evaluating the impact of a product on patients’ microbiome
- Ensuring the use of appropriate biomarkers that will correlate to an acceptable clinical efficacy endpoint
- Ensuring compliance with both pharmaceutical and GMO regulatory requirements
- Navigating the moving and multiple potential applicable regulatory frameworks as no harmonized approach is in place
Main considerations to bear in mind if you are developing a microbiome-related product:
- Microbiome products can fall under several regulatory statuses, in both the food and medicinal arenas
- Their intended use, target population, presentation form, composition, mode of action and route of administration will be key in determining the corresponding statuses
- Both drug and food pathways and associated regulatory statuses present specific benefits and constraints and will be directly related to the complexity, costs and timelines of development, and to the further marketing opportunities.
VCLS contributes to the development of products within a comprehensive range of regulatory statuses, including drugs,(biologics, ATMP), medical devices, FSMPs, Food supplements. We have a solid track of experience in various therapeutic fields including infectious diseases, inflammatory diseases, immunology (including allergies), metabolic disorders, oncology with common applications in the gastrointestinal tract, skin and gut-brain axis.
What VCLS can offer:
Early product positioning & strategical development planning
- Identification of eligible regulatory paths, within the food and therapeutic arenas, as well as in the cosmetic field
- Risk of re-qualification & identification of critical product features
- Target Product Profile (TPP) definition
- Assessment of optimal positioning based on subsequent development needs and risks
Regulatory activities implementation
- Definition of an optimal regulatory roadmap for global market entry
- Critical analysis of regulatory development plans
- Eligibility assessment & application to accelerated development pathways and authorization programs
- Support for request, preparation and participation at meetings with Competent Authorities at key steps of product development including Informational meetings, Scientific Advices, pre-IND meetings
- Author, review and submission of authorization/notifications regulatory dossiers in key global markets
Learn more about our Regulatory Strategy & Submission service
CMC, Nonclinical and Clinical development support
- Gap analysis of available CMC, nonclinical and clinical data in view of critical regulatory submissions
- Strategy support for individualized nonclinical and clinical development programs including study protocol design