FDA Sees Spike in Gene and Cell Therapy Applications

Regulatory NewsRegulatory News
| 16 February 2016 | By Zachary Brennan 

The US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) met Tuesday to discuss updates of research programs in the Office of Cellular, Tissue and Gene Therapy (CTGT), which is part of FDA’s Center for Biologics Evaluation and Research (CBER).

The research updates came as Raj Puri, MD, PhD, Director, Division of Cellular, and Gene Therapies (DCGT) at CBER told the committee that although his division has only licensed 12 products in its entire history, more than 1,530 active investigational new drugs (INDs), investigational device exemptions (IDEs) and master files are currently under review, and 106 of those INDs deal specifically with natural killer cells, which play a role in the rejection of tumors and virally-infected cells.

When asked how he plans to deal with what will likely be a backlog of applications, Puri said his division is working to meet 100% of the timelines outlined in the Prescription Drug User Fee Act (PDUFA), which requires payments from industry in exchange for an understanding of how long it will take for an FDA review (typically 10 months, though expedited reviews are six months).

He also said his division continues to plead for additional monetary and staffing resources to meet those challenges.

As far as research priorities, Puri said the division works with virology, immunology, cell and development biology, cancer biology and immunology, biotechnology (genomics, flow cytometry, proteomics) and microbiology of tissue safety:


Puri also said his division is seeing a lot of research around multipotent stromal cells right now:


Carolyn Wilson, PhD, associate director for research at CBER, explained to the committee that the center doesn’t always have the science and methodology to address new gene and cell therapies, though she’s hopeful the advisory committee may help fill some of those gaps.

She also discussed how about 20% of CBER staff are currently working as researchers/regulators, meaning they participate in advisory committees, work on policy documents, help with company inspections, work on external research and attend conferences to put them in “a unique spot to see what’s coming down the pipeline and look to classes of products to identify scientific gaps to ensure research is being used most efficiently.”

Steven Bauer, PhD, Chief of the Cellular and Tissue Therapy Branch at CBER also discussed his branch’s research on cell therapies, stem cells, adult cells and combination products, as well as gene therapies, and diagnostics and cell purification systems.

Following the open discussion and no comments during the public hearing, the advisory committee began its closed portion of the meeting, which is confidential.


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