FDA issues guidance on gene therapy

Country

United States

The US Food and Drug Administration has issued new guidance for developers of gene therapies highlighting both the complexity of manufacture as well as the difficulty in establishing duration of response in clinical trials.

In six final guidances, the agency addresses these issues as well as the application of orphan drug regulations to gene therapy. Orphan drug eligibility is an issue because many gene therapies are being developed for rare diseases where the patient populations are small.