Regulatory Actions Impacting Biologics

Three regulatory actions are coming together in an effort to move biologics, the gene therapy, and the ATMP platform forward quickly, say Susan J. Schniepp, distinguished fellow at Nelson Labs, and Siegfried Schmitt, PhD, vice president, Technical, Parexel.

Q. My company manufactures biologics, and we saw some recent actions by FDA, USP, and the EU. ow do these regulatory actions impact us as a biologics manufacturer?

A. The US Pharmacopeia (USP) has released methods for biologics, without specifications, in an effort to help biologic manufacturers, because this is a rapidly growing field. So, this should help people in terms of their chemistry, manufacturing, and controls (CMC) sections, with methods, and then they can set the specifications depending on the product they're developing.

The European Medicines Agency (EMA) announced an agreement for their Critical Medicines Act that came out in 2025. This act addresses controlling drug shortages and eliminating some of those roadblocks like suppliers, because there may be critical elements that a company needs to manufacture a biologic that might be available from only one source; if that supplier has a problem, everybody has a problem.

The other recent action is from the FDA. The agency put out a draft guidance about leveraging prior knowledge in the development of human gene therapy products incorporating genome editing. The guidance says that, instead of establishing your own holistic platform, you can reference studies and information that have been published to enhance your CMC section or your filing.

These regulatory documents are open for public comment, and companies should consider submitting their comments and/or concerns, including the USP standards.

Let’s discuss the potential intentions of the regulators in taking these actions. These products are typically life-saving products, when we look at biologics. These are products that not only the patients want to see urgently, but also the regulators are supporting the industry in getting them to market.

But not only that, there is a supply chain behind it because you cannot make everything in-house. You need suppliers. And it may be for some of these raw materials and excipients, they may be very difficult to get hold of. And some may be very difficult to transport. So, there are many different aspects to this. And some of the technical ideas are to perhaps manufacture close to where your patients are.

Because the knowledge of these products may be limited to a few people or a few firms, the FDA draft guidance is trying to expand that knowledge by allowing companies to use information that's previously been published for their product, to be able to expedite approval of these lifesaving methods.

And coming back perhaps also to the manufacturing; you could, for example, traditionally file for a product in the United States, and then you file in Europe and maybe other jurisdictions. And you may typically have a product that you start making in the US, and then you ship it over to other markets. That's not always an option with biologics, especially because cell and gene therapy products may not be conducive to such shipments.

And something that all the regulators like to see now is to have manufacture much more within the market where they are regulating, which may mean, with this distributed manufacturing, that we are probably getting away more and more from the centralized large production facilities to more smaller, closer to the patient facilities where we have shorter supply chains.

But how do we get all our suppliers to supply us to these locations rather than to just one? So, there are many aspects that need to be considered. It's important that companies provide their input to the regulators because they want to hear what is feasible and what is practical.

References

1. USP publishes product-specific emerging biologics standards for public comment. USP. Press release. June 1, 2026. Accessed July 9, 2026. https://www.usp.org/news/usp-publishes-product-specific-emerging-biologics-standards-for-public-comment
2. EMA welcomes political agreement on the Critical Medicines Act. EMA. News release. ec.europa.eu. May 12, 2026. Accessed July 9, 2026. https://ec.europa.eu/commission/presscorner/detail/en/ip_26_1017
3. FDA issues draft guidance to help accelerate cell and gene therapies for patients. FDA. Press release. June 2, 2026. Accessed July 9, 2026. https://www.fda.gov/news-events/press-announcements/fda-issues-draft-guidance-help-accelerate-cell-and-gene-therapies-patients