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Yale J Biol Med. 2017 Dec 19;90(4):683-693. eCollection 2017 Dec.

Regulatory Considerations for Gene Therapy Products in the US, EU, and Japan.

Author information

1
Department of Paediatrics, University of Oxford, Oxford, UK.
2
Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, Oxford, UK.
3
Apollo Ventures, Hamburg, Germany.
4
Nuffield Departments of Orthopaedics, Rheumatology, and Musculoskeletal Sciences, University of Oxford, Oxford, UK.
5
Oxford-UCL Center for the Advancement of Sustainable Medical Innovation, University of Oxford, Oxford, UK.
6
School of Clinical Medicine, University of Cambridge, Cambridge, UK.
7
Immusoft Corp., Seattle, Washington, USA.
8
Moorfields Eye Hospital, London, UK.
9
Oxford University Hospitals National Health Service Foundation Trust, Oxford, UK
.

Abstract

Developers of gene therapy products (GTPs) must adhere to additional regulation beyond that of traditional small-molecule therapeutics, due to the unique mechanism-of-action of GTPs and the subsequent novel risks arisen. We have provided herein a summary of the regulatory structure under which GTPs fall in the United States, the European Union, and Japan, and a comprehensive overview of the regulatory guidance applicable to the developer of GTP. Understanding the regulatory requirements for seeking GTP market approval in these major jurisdictions is crucial for an effective and expedient path to market. The novel challenges facing GTP developers is highlighted by a case study of alipogene tiparvovec (Glybera).

KEYWORDS:

ATMP; EMA; FDA; Japan; MHLW; advanced therapeutic medicinal product; gene therapy; regulation

PMID:
29259533
PMCID:
PMC5733859
[Indexed for MEDLINE]
Free PMC Article

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