01.16.19

FDA to Boost Staff to Help Handle Anticipated Increase in Cell and Gene Therapy Filings

Source: FirstWord

FDA Commissioner Scott Gottlieb said Tuesday that the agency’s “eventual goal” is to add about 50 additional clinical reviewers to the group that oversees the clinical investigation, development, and review of cell and gene therapy products. He also cited the more than 800 active cell-based or directly administered gene therapy investigational new drug (IND) applications currently on file with the agency, saying the FDA anticipates receiving more than 200 INDs per year by 2020.

“By 2025, we predict that the FDA will be approving 10 to 20 cell and gene therapy products a year based on an assessment of the current pipeline and the clinical success rates of these products,” Dr. Gottlieb said in a joint statement with Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

In a bid to “advance opportunities” in cell and gene therapy, the FDA intends to unveil additional new policy guidance and other progress made in its drug development framework this year. Specifically, the agency will collaborate with sponsors to “make maximum use of our expedited programmes,” including the regenerative medicine advanced therapy designation and accelerated approval.

The FDA is also developing a series of clinical guidance documents related to different areas of active product development, including guidance for gene therapy products for inherited blood disorders such as haemophilia, among others. In addition, the US regulator is planning to formulate a guidance document for “providing recommendations on product development issues related to the development of gene therapy products for certain neurodegenerative diseases.” According to Dr. Gottlieb, the guidance will also “propose how a more traditional approach to drug development may be more appropriate if the gene therapy creates a genetic alteration aimed at treating the symptoms of a neurodegenerative disease, or potentially altering its course by altering the expression of a protein or enzyme believed to play a role” in its progression.

Further, the FDA’s new guidance will recommend parameters for introducing manufacturing advances that make the development and application of CAR-T therapies more efficient, “without necessarily requiring costly new clinical investigations.” Moreover, it intends to develop guidance to promote the efficient development of safe and effective cell-based regenerative medicines.

“We believe these cell-based and gene therapy technologies hold tremendous promise for addressing some of the most intractable diseases,” commented Dr. Gottlieb, “but with their novelty, also comes new uncertainties and some unique, theoretical risks. Our efforts are aimed at helping innovators proactively address these potential risks, while we outline a modern and efficient pathway for the continued development of these innovations.”

Separately, Gottlieb indicated that some drug inspections halted during the US government shutdown would resume on Tuesday.

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