USA – CBER Plans Draft Guidance on ‘Sameness’ of Gene Therapies Under Orphan Drug Regulations

The US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) has updated its list of 2019 guidance and added a new draft on interpreting “sameness” of gene therapy products under the orphan drug regulations.

The new draft on sameness, under the category of Tissues and Advanced Therapies, is expected in addition to the finalization of six guidance documents on gene therapies released last July, final guidance from February on expedited programs for regenerative medicines and the evaluation of devices with regenerative medicines, and final guidance from March on CBER’s use of standards in regulatory submissions.

But FDA’s discussion of sameness in the context of gene therapies and orphan exclusivity will be highly anticipated, as about 70% of gene therapies are seeking to treat rare diseases and as the complexity of gene therapies may make it difficult for the agency to set a precedent…