Regulatory Considerations for Gene Therapy Products in the US, EU, and Japan.
Yale J Biol Med
; 90(4): 683-693, 2017 12.
Article
en En
| MEDLINE
| ID: mdl-29259533
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).
Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Terapia Genética
Tipo de estudio:
Guideline
Límite:
Humans
País/Región como asunto:
America do norte
/
Asia
Idioma:
En
Revista:
Yale J Biol Med
Año:
2017
Tipo del documento:
Article
País de afiliación:
Reino Unido