FDA’s positive reinforcement

How FDA’s gene therapy guidances reinforce the paths of the most advanced candidates

Companies are still sifting through a half dozen gene therapy guidance documents issued by FDA, but so far it appears the recommendations support the paths taken by some of the most advanced clinical candidates for hemophilia and retinal disorders. The new documents also could eliminate or reduce the requirements for follow-up studies for some products.

Three of the six draft guidances issued July 11 cover development of gene therapies to treat rare diseases, retinal disorders and hemophilia.

The remaining three, related to chemistry, manufacturing and controls (CMC); long-term follow-up studies; and manufacturing and follow-up for retroviral-based therapies, are updates to existing guidances that, once finalized, will replace outdated documents.

In a prepared statement issued with the guidance documents, FDA Commissioner Scott Gottlieb said an inflection point has been reached in the development of gene therapies. “We expect this field to continue to expand, with the potential approval of new treatments for many debilitating diseases,” he said.

The guidance is “intended to serve as the building blocks of a modern, comprehensive framework for how we’ll help to advance the field of gene therapy, while making sure new products meet the FDA’s gold standard for safety and effectiveness,” Gottlieb said.

Comments on the six draft guidance documents are due Oct. 10.

The agency chose rare diseases, hemophilia and retinal disorders because of the sheer number of candidates in the clinic to treat these indications. BioCentury’s BCIQ database shows one in vivo gene therapy on the

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