Live Biotherapeutic Products, A Road Map for Safety Assessment

Area of Science:

• Microbiology and Immunology
• Pharmacology and Drug Development
• Regulatory Science

Background:

• The host-microbiota relationship is increasingly recognized for its therapeutic potential.
• Live Biotherapeutic Products (LBPs) are defined as medicinal products requiring authorization in the EU.
• Current pharmaceutical regulations present challenges for LBP commercialization due to their unique characteristics.

Purpose of the Study:

• To provide a roadmap for risk analysis specific to Live Biotherapeutic Products (LBPs).
• To address the challenges in safety assessment for LBPs compared to conventional medicinal products.
• To guide the design of non-clinical and early clinical development programs for LBPs.

Main Methods:

• Detailed risk analysis considering the specific characteristics and mode of action of LBPs.
• Review of existing pharmaceutical guidelines for biological products.
• Proposal of solutions for designing relevant non-clinical and First-in-Human (FIH) trials.

Main Results:

• Identification of unique risks associated with LBPs and their interactions with patients.
• Development of a structured approach to LBP risk assessment.
• Recommendations for tailored non-clinical and clinical trial designs to evaluate LBP safety.

Conclusions:

• A specific risk analysis framework is crucial for the safe development of LBPs.
• Understanding LBP-specific safety profiles is essential for regulatory approval.
• The proposed roadmap facilitates the design of appropriate studies for LBP clinical evaluation.