Pharma

Nanomedicine, the FDA: Developing science on a case-by-case basis

Nanomedicine offers the promise of new drugs with targeted delivery and fewer side effects, but […]

Nanomedicine offers the promise of new drugs with targeted delivery and fewer side effects, but the companies developing such medicines encounter this challenge: The U.S. Food and Drug Administration has no official definition of nanotechnology.

In fact, much of the guidance that could help nanomedicine companies remains unwritten so far. All of the products that contain nanomaterials go through the FDA’s traditional regulatory pathways for small molecule drugs.

The still-developing regulations for nanomedicines was the topic of the latest NanoBio Executive Roundtable, a series of panel discussions presented by the Center of Innovation for Nanobiotechnology, or COIN. Katherine Tyner, a chemist in the FDA’s division of drug safety research, represented the FDA at the latest panel discussion this week, which was held at the Research Triangle Park Foundation’s headquarters in RTP. Tyner, who qualified her comments with the disclaimer that nothing she said should be interpreted as FDA policy, said that while the rules and definitions for nanomedicines are still being developed, companies could find some guidance in the rules for other medicines. Many biological products, for example, fall into the same size range as nanomedicines. While biologics aren’t nanotech drugs, they can share some of the stability and manufacturing issues that nanomedicines have. For example, the FDA does have biologics guidance on stability — a compound’s shelf life and the effects of conditions such as temperature and humidity.

“I got in trouble when I called it a long guidance — it’s 80 pages long,” Tyner said. “I’m told it’s a comprehensive guidance.”

The absence of rules has not prevented companies from working on nanomedicines. Maryland nanomedicine company CytImmune Sciences has developed a way to use nanoparticles of gold for the targeted delivery of chemotherapy to cancer tumors. CEO and co-founder Larry Tamarkin explained that the promise of his company’s nanomedicine is the ability to use less of a drug because with nanotechnlogy, more of the drug will get to the site of the disease. As a result, patients should experience fewer side effects. But Tamarkin said the Achilles’ heel of nanomedicine is manufacturing. Nanomedicine manufacturing is complex and the FDA will want to see manufacturing done to certain standards.

“If you can’t do that, it’s going to be a hard slog,” Tamarkin said.

Some attendees from the Triangle’s scientific community pressed Tyner on the proper approach to presenting new nanomedicines to the FDA. One asked Tyner whether a nanotech product that had multiple functions should seek approval of its functions all at once or one at a time. Tyner’s response? The FDA will look at nanotechnologies on a case-by-case basis. It’s a less than ideal answer, but for now, it’s the one that will have to do.

It takes years for a drug to be developed. Tamarkin said CytImmune needed more than a decade “to go from bench to bedside.” And the company’s lead candidate is still years from completing clinical trials. It may also take years for the FDA to draft nanotechnology guidelines. While no company wants to wait, companies who are wondering just how to approach the FDA might benefit from watching and waiting.

Nancy Teague, executive director of regulatory affairs for Wilmington, North Carolina clinical research organization PPD (NYSE:PPD), offered her perspective as someone working in a industry, as well as someone who has worked on the other side. Teague spent part of her 30-year career in life sciences working for the FDA. Teague said that companies might benefit from coming to the FDA after the first few nanomedicine companies have gone through the process. The experiences of those companies could pave the way for others. CytImmune is one of those companies that the FDA has looked at on a case-by-   case basis and Tamarkin said he has had no problems.  It’s not ideal and it’s far from “comprehensive guidance.” But that’s what happens when breakthrough medical technology moves faster than the ability of regulators to keep up with it.

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