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. 2021 Aug 11:8:716266.
doi: 10.3389/fmed.2021.716266. eCollection 2021.

Entering First-in-Human Clinical Study With a Single-Strain Live Biotherapeutic Product: Input and Feedback Gained From the EMA and the FDA

Jeanne-Céleste Paquet  1 , Sandrine P Claus  1 , Magali Cordaillat-Simmons  2 , Wilfrid Mazier  1 , Georges Rawadi  1 , Laure Rinaldi  1 , Frédéric Elustondo  1 , Alice Rouanet  2
Affiliations

Entering First-in-Human Clinical Study With a Single-Strain Live Biotherapeutic Product: Input and Feedback Gained From the EMA and the FDA

Jeanne-Céleste Paquet et al. Front Med (Lausanne). .

Abstract

During the last decade, a plethora of novel therapies containing live microorganisms as active substance(s) has emerged with the aim to treat, prevent, or cure diseases in human beings. Both the Food and Drug Administration (FDA) and the European Directorate for the Quality of Medicines and Health Care (EDQM) codified these biotherapies as Live Biotherapeutic Products (LBPs). While these innovative products offer healthcare opportunities, they also represent a challenge for developers who need to set the most suitable designs for non-clinical and clinical studies in order to demonstrate a positive benefit/risk ratio through relevant quality, safety, and efficacy data that are expected by the drug competent authorities. This article describes how YSOPIA Bioscience, supported by the Pharmabiotic Research Institute (PRI), addressed the regulatory challenges during the early development phase of their single-strain LBP, Xla1, in order to obtain the necessary authorizations to bring this drug to the clinical stage.

Keywords: European Medicines Agency; FIH clinical trial; Food and Drug Administration; LBP; Live Biotherapeutic Products; Microbiome; regulatory science.

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Conflict of interest statement

JCP, SC, WM, GR, LR, and FE are employees of YSOPIA Bioscience. MC-S and AR are employees of the Pharmabiotic Research Institute.

Figures

Figure 1
Figure 1
YSOPIA risk management plan to manage potential clinical infection applied during phase 1 trial.
See this image and copyright information in PMC

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