The draft guidance, which focuses on the non-clinical and clinical aspects of gene therapies being developed, was released last week by the European Medicines Agency (EMA) and comes more than two years after the European regulatory body approved its first gene therapy medicinal product (GTMP) - UniQure’s Glybera, for patients with the rare metabolic disorder Lipoprotein Lipase Deficiency.
The guidance intends to help what the EMA says are often small-sized companies or academic bodies conduct robust development and understand the necessary regulatory steps needed to bring such therapies through the regulatory process.
The agency has focused on the quality, safety and efficacy requirements of GTMP development and manufacture, and both the non-clinical and clinical studies needed to be undertaken. Furthermore, it takes into account newer gene therapy technologies such as microRNA and gene silencing/knockdown being used by drugmakers.
“The draft guideline takes stock of the experience gained with gene therapies in recent years through scientific advice, the classification of advanced therapies and marketing-authorisation applications, and addresses issues observed during these procedures,” the agency stated.
“This includes in particular critical quality issues in a number of applications involving gene therapies which have an impact on the validity of the non-clinical and clinical studies.”
In particular the EMA emphasises the fact that across all areas “high quality standards need to be reached before non-clinical and clinical studies can be started.”
Drugmakers have three months to comment on the guidance, which – once finalised – will replace the current 2001 note for guidance on gene therapies.