The Near Future of Gene Therapy Regulation

The Clinical Trials and Regulatory Affairs Committee - July 02, 2018

In a meeting with ASGCT organizational partner Research America, Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER), discussed the upcoming FDA guidance documents— retinal disorders was added to CBER’s Guidance Agenda earlier in the week—and elaborated on the agency’s interaction with their European counterparts in relation to gene and cell therapy.

Marks remarked on a number of important issues facing the field, but perhaps the most prescient of them are the six gene therapy guidance documents the FDA plans to release in the near future.

According to Marks, all six documents are in an “advanced stage” of development and are likely to be publically issued this summer.

The guidance documents will be related to clinical pathways for retinal disorders, and hemophilia.  FDA may also consider guidance for some other diseases that are less common. The final three are related to manufacturing and long-term safety.

Marks spoke extensively about his and the FDA’s important relationship with their European counterparts with the European Medicines Agency (EMA) and the European Commission (EC). At a previous meeting in Brussels, Belgium, all three organizations re-affirmed their collaborative nature in a number of fields including advanced therapies (ATMPs) like gene therapy.

“Regulators on both sides of the Atlantic are actively supporting the development of these novel medicines and are facing similar regulatory challenges,” an EC news release from the June meeting reads. “The parties therefore agreed to encourage early parallel scientific advice and to further strengthen the existing ‘cluster’ on ATMPs with a view to develop common scientific approaches on the regulation of these medicines, that could best facilitate their preclinical and clinical development, and the way data collection on these medicines can be optimised after authorisation.”

The FDA will also collaborate with the European Directorate for the Quality of Medicines.

Additional Highlights

Marks also touched on a number of regulatory and research issues throughout the Research America talk:

On CBER INTERACT (INitial Targeted Engagement for Regulatory Advice on CBER ProducTs) Meetings

  • CBER is updating the process to improve meeting consistency. The new INTERACT website will be updated with more information about anticipated timelines and expectations during the meeting. The INTERACT meetings will replace the pre- pre-investigational New Drug (IND) meetings and allow academics and companies to get CBER feedback on regulatory expectations earlier in the development process.

On Manufacturing

  • If sponsors expect to see efficacy in Phase I for products with easily demonstrable efficacy, then FDA recommends developing a process that can be easily transferable to GMP.  In short, start early with scalable manufacturing processes if you expect early efficacy.

On Regenerative Medicine Advanced Therapy (RMAT) designation

  • FDA handles RMAT designation similarly to its breakthrough therapy designation with the key difference being different qualifying criteria for clinical preliminary evidence. Breakthrough therapy designation requires substantial improvement over existing treatments, while RMAT designation is for drugs with the potential to address unmet needs for treating a disease or condition.
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