Cell and gene therapies: FDA expects 10 to 20 approvals per year by 2025

by Dan Stanton

The US FDA predicts it will receive more than 200 regenerative INDs per year from 2020 and will add 50 additional staff to review these products.

There has been “a large upswing in the number of investigational new drug (IND) applications” for cell and gene therapies received by the US Food and Drug Administration (FDA), commissioner Scott Gottlieb and Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) said in a statement yesterday.

The Agency has so far received over 800 INDs for proposed regenerative medicines and predicts from next year industry will submit upwards of 200 INDs for review per year.

“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.”

As such, the Agency says it is planning to introduce a series of actions, including additional new policy guidance, to aid industry and expedite products to market.

As well as working with sponsors to use the FDA’s current expedited programs – including regenerative medicine advanced therapy (RMAT) designation – it is planning a series of clinical guidance documents related to different areas of active product development.

And to cope with increased submissions, the Agency says it will add “about 50 additional clinical reviewers to the group charged with overseeing the clinical investigation, development, and review of these products.”

The FDA says these activities reflect a turning point in the development of these new technologies and their application to human health and compared it with the shift in development of antibody drugs in the late 1990s, leading to the mainstreaming of monoclonal antibodies in the present era.

July guidance
This latest information from the FDA comes off the back of a series of draft guidance documents issued last July focused on gene therapies for specific disease areas and the manufacture of gene therapies.

In a video published ahead of next week’s Phacilitate conference in Miami, Florida, Anthony Davies, founder and CEO at Dark Horse Consulting, gave his view on these regulatory documents.

“The field of cell and gene therapy has moved faster in the last several years than in the last several decades,” he said. “This is acknowledged by the Agency in these guidances but we feel it is not yet reflected in actual content.

“We are still looking for more specific guidance and we would like to see more specialized guidance to focus on pure play cell therapies which are not gene modified, gene-modified cell therapies and pureplay gene therapies wherein the cell is relegated to a manufacturing platform rather than being the product itself. We feel that each of these three categories deserve a specific guidance of its own, and we very much hope the Agency will follow through with such guidances in the near future.”