Follow-on Biologics Workshop
Posted July 01, 2009
The New York Academy of Sciences, in conjunction with the FDA and the National Institute for Standards and Technology (NIST), held a workshop on December 12–14, 2005 that focused on the scientific issues involved in assessing the similarity of follow-on protein products. At the conference, scientists from academe, industry, and government surveyed the scientific issues surrounding protein structure and function, focusing on specific analytical methodologies for characterizing protein products.
The first half of the conference focused on the challenges posed by the complexities of protein structure. Day one covered primary, secondary, and tertiary structure, and analytical methods for characterizing those structures. On the first half of day two, speakers discussed quaternary structure, protein-protein interactions, and protein aggregates. Speakers from FDA and NIST introduced these sessions, laying out the scientific and regulatory challenges presented by each protein structure element. During the second half of day two, several speakers discussed the impact of processing on protein properties.
Biotechnology Industry Organization (BIO)
BIO has collected their comments on follow-on biotechnology product regulation as full-text pdf files here.
EMEA/DIA Joint Workshop on EMEA New Guidelines for Development and Approval of Biosimilars
Program for a December 2005 conference on European regulation of follow-on biologics (PDF, 368 KB).
European Medical Association Guidelines
Emerging Biopharmaceutical Enterprises, an organization of European pharmaceutical companies, has posted documents from the European Medical Association here related to the regulation of follow-on biologics, called "biosimilars" in Europe. See in particular:
- Guideline on similar biological medicinal products (PDF, 194 KB)
- Guideline on similar biological medicinal products containing biotechnology-derived proteins as active non-clinical and clinical issues (PDF, 89 KB)
- Guideline on similar biological medicinal products containing biotechnology-derived proteins as active substance: quality issues (PDF, 203 KB).
FDA Center for Biologics Evaluation and Research
Web site of the FDA division responsible for regulating blood-derived biological products.
FDA Center for Drug Evaluation and Research
Web site of the FDA division responsible for regulating most protein products.
The Law of Off-Patent Biopharmaceuticals
A statement by Senator Orrin Hatch on steps Congress must take to provide the regulatory framework for follow-on biologics.
PhRMA Comments to the FDA on follow-on biologics (PDF, 805.1 KB)
PhRMA (The Pharmaceutical Research and Manufacturers of America) advocates for public policies on behalf of pharmaceutical/biotechnology research companies.
Scientific Considerations Related to Developing Follow-On Protein Products (February 2005)
Collected slide presentations for a February 14-16, 2005, FDA Meeting
Scientific Considerations Related to Developing Follow-On Protein Products (September 2004)
Slide presentations for the earlier, September 14-15, 2004 FDA Meeting. This online docket also contains a transcript of the meeting.
Journal & Magazine Articles
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Kurt A. Brorson, PhD
Kurt Brorson is a staff scientist in CDER's division of monoclonal antibodies, Office of Biotech Products. Kurt Brorson received a BA in biology from the University of Chicago in 1984 and PhD in molecular biology from the California Institute of Technology in 1990. After a two-year postdoctoral fellowship at the NIH, he joined the FDA in 1992 as a staff fellow and was converted to a staff scientist in 1999. In addition to review and policy activities, he conducts research on viral safety issues associated with biotechnology products.
David Bunk, PhD
David Bunk received his doctorate in chemistry from Texas A&M University in 1992. He then was awarded a National Research Council Postdoctoral Fellowship at the National Institute of Standards and Technology. After finishing his postdoctoral fellowship, he continued his research at NIST in the analytical chemistry division.
His research interests at NIST focus on the application of chromatographic and mass spectrometric techniques to the analysis of proteins. Recently he has been investigating the use of proteomics method for the quantification of clinically-relevant proteins in serum, specifically cardiac troponin I, c-reactive protein, and B-type natriuretic peptide.
Bunk is a member of the American Society for Mass Spectrometry and the American Association for Clinical Chemistry and actively serves on committees for the American Association for Clinical Chemistry, the International Federation of Clinical Chemistry, and the Clinical Laboratory Standards Institute.
Barry W. Cherney, PhD
Barry Cherney is deputy director at the division of therapeutic proteins in the office of biotechnology products at FDA's Center for Drugs Evaluation and Research (CDER). His regulatory duties have included oversight of reviews for INDs and BLAs for recombinant and naturally derived proteins, product reviewer training, and development of regulatory guidance to address scientific issues related to characterization, manufacture, and control of specified and naturally derived protein products.
Cherney obtained his PhD in biology from Case Western Reserve University. His dissertation work involved the purification and characterization of the nuclear enzyme, Poly (ADP-ribose) synthetase. In 1986, he began postdoctoral training in the department of biochemistry and molecular biology at Georgetown University where he worked on molecular approaches toward defining the structure/function of this enzyme. He joined CBER in 1991 as a senior staff fellow in the division of hematological products and in addition to his regulatory duties, conducted research on the molecular mechanisms leading to tumorigenesis. Since 1998, Cherney has held positions as a full time regulatory reviewer, an expert biologist and became deputy director of the division of therapeutic proteins in March 2001. He was FDA's topic lead expert on ICH Q5E for comparability of biotech products and has been involved with issues concerning the comparison of protein products throughout his FDA career.
Curtis Meuse, PhD
Curtis Meuse is a scientist in the biochemical science division of Chemical Science and Technology Laboratory at the National Institute of Standards and Technology (NIST). He received his PhD in chemistry at the University of Massachusetts, Amherst studying ultra-thin polymer layers using infrared spectroscopy. As a postdoctoral fellow in the Laboratory of Chemical Physics at the National Institutes of Health, he synthesized specifically fluorinated phospholipids and studied their nano-domain structure using Raman spectroscopy. In 1995, he was hired as part of a NIST-wide initiative to develop expertise in the development and characterization of new biomimetic materials. In the Biomolecular Materials Group, he developed methods and models for the characterization of the molecular structure of cell membrane components organized on surfaces using neutron reflectivity and optical techniques. In addition, a state-of-the-art infrared spectroscopic ellipsometer was built and methods for the analysis of spectroscopic data to determine the thickness, optical constants and molecular structure of various cell monolayer and bilayer constructs were developed.
Currently in the biospectroscopy group, Meuse’s work is focused on protein conformation measurements. The goals of his work are to develop and standardize methods to characterize the biologically active state, to allow the measurement of structural changes, and to characterize physical processes that contribute to biological inactivation of proteins such as aggregation and macromolecular association/dissociation. For example, infrared methods have been developed to quantify protein structural stability and protein binding interactions by quantifying the extent of the exchange of deuterium for hydrogen in the amide bonds of proteins in solutions, as solids, in membranes or immobilized on surfaces. In addition, collaborations with the United Kingdom’s National Physics Laboratory are underway on a pilot study for the standardization of biomolecular circular dichroism measurements under the auspices of the International Committee for Weights and Measures.
Emily Shacter, PhD
Emily Shacter is chief of the laboratory of biochemistry in the division of therapeutic proteins of the Office of Biotechnology Products, Office of Pharmaceutical Science, CDER. She received her PhD in biochemistry from Johns Hopkins University in 1982, carried out basic cell regulation and cancer research at the National Institutes of Health for 12 years, and then joined the FDA in 1994.
Shacter oversees the review of INDs and BLAs for novel therapeutic proteins covering a wide range of clinical indications, including cancer, hematopoiesis, tissue repair and remodeling, thrombosis and thrombolysis, inflammatory disease, and bioterrorism. She is engaged in establishing CDER policy regarding product manufacture and characterization and performs inspections of biotechnology manufacturing facilities.
Shacter also runs an active laboratory research program to support the scientific review of therapeutic proteins that have wide-ranging mechanisms of action and unique biological and physicochemical characteristics. Her laboratory research focuses on studying the cytotoxic mechanisms of cancer chemotherapy drugs and understanding how elements of the immune system, such as oxidants and activated phagocytes, influence killing and clearance of dying cells. Her research expertise in the area of protein oxidation is applied regularly to the review of protein therapeutics.
Jon Amster, PhD
François Baneyx, PhD
Adrian Bristow, PhD
Karin D. Caldwell, PhD
John F. Carpenter, PhD
Kathleen A. Clouse, PhD
Charles Cooney, PhD
Steve Cramer, PhD
Erik J. Fernandez, PhD
Ewa Folta-Stogniew, PhD
Daron I. Freedberg, PhD
William Hancock, PhD
Sarah W. Harcum, PhD
Donald Hunt, PhD
Igor Kaltashov, PhD
Steven Kozlowski, MD
Food and Drug Administration
Office of Biotechnology Products
Tom Laue, PhD
Laureen Little, PhD
Roger E. Marchant, PhD
Russ Middaugh, PhD
Keith Oberg, PhD
Alfred Mann Foundation
Vernon Reinhold, PhDUniversity of New Hampshire
Department of Chemistry
email | web site | publications
Nadine M. Ritter, PhDBiologics Consulting Group, LLC
email | web site | publications
C. Jane Robinson, PhD
National Institute for Biological Standards and Control
Division of Immunology and Endocrinology
Amy Rosenberg, MD
Food and Drug Administration
Division of Therapeutic Proteins
Frederick Schwarz, PhD
Tim D. Veenstra, PhD
Keith Webber, PhD
Angelo DePalma is a freelance writer based in Newton, New Jersey. In 1984, he received a PhD in chemistry from the State University of New York, Stony Brook. His work appears in a dozen pharmaceutical industry trade magazines, and he is the author of a bestselling book on vitamins and supplements.