USA – FDA Finalizes 6 Gene Therapy Guidances, Unveils a New Draft

The US Food and Drug Administration (FDA) on Tuesday finalized six guidance documents on gene therapy development and released a new draft guidance on interpreting the sameness of gene therapies under the orphan drug regulations.

The guidance release comes as more than 900 investigational new drug applications are ongoing for gene and cell therapy clinical studies, and as FDA has struggled to hire enough experts to tackle the related challenges. The release of the six final guidances and the new draft were expected as FDA’s list of guidance documents from last July explained.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, explained: “Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.”…