Regulatory Considerations for Gene Therapy Products in the US, EU, and Japan.
Year: 2017
Type: article
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).
Source: PubMed Central
Authors: Celine-Lea Halioua-Haubold, James G Peyer, James A Smith, Zeeshaan Arshad, Matthew Scholz, David A Brindley, Robert E MacLaren
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W2782766423