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Regulatory Aspects and Approval of Biopharmaceuticals for Mucosal Delivery: Quality, Toxicology, and Clinical Aspects

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Mucosal Delivery of Biopharmaceuticals

Abstract

Regulation of products for mucosal application has many points in common with products for other routes but also some that are unique. Identifying regulatory pitfalls is of tremendous importance early in development as this saves time, money, and nerves. There is a huge amount of regulatory advice available from the different players in the field (governments and related organizations) that is partly very general or exceptionally specific. This chapter is meant to provide guidance on what to find, where and who to ask if certain questions arise. It also provides a description of common and known problems differentiated by the three major parts: quality, preclinical, and clinical development. Finally, special key points focusing on pediatric use, use of genetically modified organisms or products classified as advanced therapy medicinal products are discussed.

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Abbreviations

BA:

Bioavailability

BE:

Bioequivalence

EMA:

European Medical Agency

ERA:

Environmental risk assessment

FDA/CBER:

Food and Drug Agency/Center for Biologics Evaluation and Research

FBS:

Fetal bovine serum

GMO:

Genetically modified organism

ICH:

International Conference of Harmonization

IMP:

Investigational medicinal product

MABEL:

Minimal anticipated biological effect level

NOAEL:

No observed adverse effect level

PCR:

Polymerase Chain Reaction

PD:

Pharmacodynamics

PDCO:

Pediatric Committee

Ph. Eur.:

European Pharmacopoeia

PIP:

Pediatric investigation plan

PK:

Pharmacokinetics

USP:

United States Pharmacopoeia

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Authors and Affiliations

  1. Paul-Ehrlich-Institut, Federal Institute for Vaccines and Biomedicines, Paul-Ehrlich-Straße 51–59, 63225, Langen, Germany

    Karen Brigitta Goetz, Yuansheng Sun, Katrin Féchir, Evelyne Kretzschmar & Isabel Buettel

Authors
  1. Karen Brigitta Goetz
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  2. Yuansheng Sun
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  3. Katrin Féchir
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  4. Evelyne Kretzschmar
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  5. Isabel Buettel
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Correspondence to Karen Brigitta Goetz .

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Editors and Affiliations

  1. IINFACTS - Instituto de Investigação e Formação Avançada em Ciências e Tecnologias da Saúde, Instituto Superior de Ciências da Saúde-Norte, CESPU, Gandra, Portugal

    José das Neves

  2. NEWTherapies Group, INEB – Instituto de Engenharia Biomédica, Porto, Portugal

    Bruno Sarmento

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Goetz, K., Sun, Y., Féchir, K., Kretzschmar, E., Buettel, I. (2014). Regulatory Aspects and Approval of Biopharmaceuticals for Mucosal Delivery: Quality, Toxicology, and Clinical Aspects. In: das Neves, J., Sarmento, B. (eds) Mucosal Delivery of Biopharmaceuticals. Springer, Boston, MA. https://doi.org/10.1007/978-1-4614-9524-6_25

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