Dr Peter Marks, head of the US Food and Drug Administration (FDA)'s Centre for Biologics Evaluation and Research, has said that the agency needs use accelerated approval to advance gene therapies for rare disease.
'The FDA views gene therapy as an excellent opportunity to expedite the delivery of potentially life-saving therapies to patients with rare diseases,' said Dr Marks in a statement.
The FDA will support using biomarkers as substitutes for other biological indicators in gene-therapy clinical trials to help achieve accelerated approval.
Dr Marks also indicated that there may be some level of uncertainty on the side-effects over long-term and safety during administration to achieve accelerated approval. He also added that post-marketing tools such as safety monitoring and the possible use of extra clinical trials are going to be key.
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