FDA expands Guideline Activities around Gene Therapies

Recommendation

7/8 May 2024

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Regulatory Requirements and Practical Implementation

When it comes to the development of new therapies, gene therapies play an important role. This has already shown in the recent past through the increasing number of guidelines and recommendations issued by the authorities (as already reported in the news on the increasing importance of gene therapies).

Recently, the FDA has announced a whole series of guidelines for gene therapies. In the press release, the FDA explained: "One of the most important steps the FDA can take to support safe innovation in this field is to create policies that provide product developers with meaningful guidance to answer critical questions as they research and design their gene therapy products."

So far, the FDA has approved four such products. However, there are more than 900 applications for clinical studies in this area. This shows the increasing importance of such therapies in general and especially in the treatment of rare genetic disorders or autoimmune diseases.

The currently published six final guidance documents and a draft guidance document from the FDA provide recommendations for development and manufacturing and offer guidance for anyone focusing on gene therapy products to treat specific diseases. Comments from various stakeholders and expert groups have been taken into account. These guidelines thus represent an important step towards the modern development of gene therapy products. They also aim to ensure that the necessary safety and efficacy requirements or standards are defined and met for such advanced therapies. The FDA does, however, allow for such therapies:

"The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality. In addition, the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs, such as questions about the durability of response, and these questions often can't be fully answered in pre-market trials of reasonable size and duration."

Further information can be found in the FDA press release: "FDA Continues Strong Support of Innovation in Development of Gene Therapy Products".

In detail, the FDA's current guidance programme includes the following documents:

• Draft Guidance: Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations 
• Final Guidance: Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) 
• Final Guidance: Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up 
• Final Guidance: Long Term Follow-Up After Administration of Human Gene Therapy Products 
• Final Guidance: Human Gene Therapy for Hemophilia 
• Final Guidance: Human Gene Therapy for Retinal Disorders 
• Final Guidance: Human Gene Therapy for Rare Diseases