CGT CMC: Plan ahead, avoid comparability studies says Novartis

Making changes to the manufacturing process for cell and gene therapies is possible but cumbersome due to the need to perform comparability studies, says a Novartis veteran.

Speaking at the Cell & Gene Therapy Bioprocessing & Commercialization conference – part of Biotech Week Boston – this week, Novartis’ Sergio Fracchia advised developers of advanced therapies to think about their manufacturing needs several years in advance to avoid future pitfalls.

Specifically, Fracchia – who serves as Global regCMC Director, Cell and Gene Therapy at the Swiss biopharma – spoke of advanced therapy medicinal product (ATMP) raw materials.

Sergio Fracchia, Global regCMC Director, Cell and Gene Therapy at Novartis spoke at Biotech Week Boston

Ensuring their quality and the manufacturing process involved is of the highest standard from the start of the development process, will save time and effort later, said Fracchia, and removes the “difficult and cumbersome” need to undergo comparability studies.

When improving the quality of your raw materials during development “you can switch from one to the other but this is not for free. Changes in critical materials like those of biological origin – human serum for example – but even recombinant need comparability studies,” he told delegates.

“And comparability studies are not easy to be performed, and not easy to be evaluated because at the end your product is a cell-based product which is not comparable by definition and so the assessment of the comparability is not straight forward.”

Because of this he advised ATMP developers to look ahead “two, three, four years’ time” to avoid changes and the necessary comparability studies.

Raw and starting materials

Both Fracchia and Scott Burger, Principal of Advanced Cell & Gene who spoke in the same session, stressed that there is no standard terminology as yet regarding materials used in cell and gene therapies.

In Europe, raw materials are defined as component, reagents or materials exerting effects on but not intended to be part of the final cell product. Whether chemically synthesized or from an animal, human or recombinant origin, they do have a direct impact on the quality, safety and efficacy of the final product and require to be manufactured under GMP conditions.

Starting materials, meanwhile, are defined as those being directly used in manufacture and intended to be part of the final product.

In the US, the definition of raw materials includes the cell and tissue material, along with ancillary reagents not intended to be present in the final product, and excipients.

Related news: Garbage in, garbage out: Raw material quality crucial for cell & gene therapies

Leave a Reply