2:21 PM
Mar 22, 2019
 |  BioCentury  |  Regulation

Manufacturing new modalities

What FDA and industry are doing to improve manufacturing of cell and gene therapies

Cell and gene therapies won’t enter the mainstream of medicine until manufacturing processes become far more efficient, according to FDA Commissioner Scott Gottlieb and FDA Center for Biologics Evaluation and Research Director Peter Marks. The agency hopes to play a major role in making manufacturing improvements possible, they told BioCentury.

In the U.S., groups like the Alliance for Regenerative Medicine (ARM) and Friends of Cancer Research are working with FDA, manufacturers and academic investigators on standards and new regulatory approaches to topics like comparability and standards that could lay the groundwork for industrializing the manufacturing of new modalities.

Regulatory authorities in Europe and Japan are also collaborating with industry and academic investigators to streamline manufacturing. There are concerns in Europe, however, that a fractured regulatory structure is holding back regenerative medicine (see “Regulating Regeneration in Europe”).

Creating regulations that both enable progress and ensure safety raises unique challenges in cell and gene therapies because the products often reach the market with limited clinical testing, and can be incorporated into patients’ bodies in ways that are long lasting, even permanent.

Regulators’ responses to rapidly evolving manufacturing technologies are especially important for CAR T therapies.

Unprecedented efficacy in treating some deadly cancers has prompted drug companies to speed CAR T products to market. Manufacturing technologies are advancing even more quickly than clinical progress, but they may not be adopted because sponsors want to avoid triggering requirements to conduct additional trials or possibly recapitulate entire development programs.

A wave of regenerative medicine products is close to commercialization and manufacturing hasn’t kept up, Gil Van Bokkelen, CEO and chairman of Athersys Inc., told BioCentury. “Commercial-scale manufacturing for the regenerative medicine sector as a whole is hitting a wall.”

One of the challenges that particularly concerns Marks is reducing the friction involved in moving from early stage academic research to larger clinical trials and commercial products (see “Academia’s Manufacturing Problem”).

One of Marks’ ideas is for FDA and industry to create “cookbook” approaches to viral vector manufacturing that would make it easier to transition gene therapies developed...

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