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  5. Daniela Verthelyi, M.D., Ph.D. - Transcript
  1. Regulatory Science in Action

Daniela Verthelyi, M.D., Ph.D. - Transcript

Developing methods used for the regulatory review of protein-based drugs

Dr. Verthelyi: I’ve been at the FDA for almost 20 years and I came right out of grad school. And I was attracted to the idea of doing research in a very applied fashion, and doing the regulations that directly affects public health.

Text Slide:

Daniela Verthelyi, M.D., Ph.D.

Chief, Laboratory of Immunology

Division of Biotechnology Review and Research III

Office of Pharmaceutical Quality

Office of Biotechnology Products

Dr. Verthelyi: The Office of Biotechnology Products is in charge of reviewing biologics. Biologics are the kind of drugs that are made by living cells, so whether they’re living cells that we have, that we derive from an animal or a plant, or they’re living cells that come from a bioreactor where you grow the cells, they’re still living cells making these proteins, and those are the kinds of products that we regulate.

These proteins are purified and formulated so that people can take them. And we look at the chemistry, the manufacturing, the stability of these proteins. My group in particular looks at whether these proteins can induce an immune response in the host.

You don’t want the body to make a response and eliminate that protein, you want that protein to be effective.

In our lab we develop a lot of new methods. Not just animal models, but also lab based methods that can be used for regulatory purposes. That will help us all get more drugs to more people in a more accessible way.

A lot of times your pharmacists will ask you, would you like the original or would you like the generic. That happens to us all when we go to the pharmacy. It used to be that these other types of products that are more complex, these therapeutic proteins we couldn’t have the generic type because they were just so complex, they were so difficult to characterize that it couldn’t be done.

Now science advances and our capacity to regulate the science advances, and now we have biosimilars. So we are starting to have these very complex products that can be manufactured by a different company, but use a lot of the safety and efficacy data that was generated by the first company.

There is one project that I’m particularly excited about, and that has to do with a disease called Cutaneous Leishmaniasis, which is caused by a small parasite transmitted by a little fly. Once it’s embedded in the skin, it causes lesions that can be deforming, and get super infected or painful. And we’re working in partnership with WHO and with some brilliant scientists around the world to develop new therapeutics for this disease which has no really good therapeutics available. If we are successful, it should improve the life of millions of people around the world.

So we are always looking into the future to try to understand what are going to be the regulatory priorities and what kind of science we need to develop in order to regulate the upcoming products.

Top-notch science is going on daily at the FDA to support those regulatory decisions. Supporting that science is critical, because it allows us really to advance medicine.

Working at the FDA really affords me some incredible opportunities of working together with companies and developing therapeutic products that are going to really affect the lives of the patients. And it really also affords me incredible freedom to explore the science. So it’s that combination that really makes this job a really great one.

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