Open Access Thesis
Master of Science (MS)
Irwin Martin, Ph.D.
Jean Rowan, MD, MS.
Any new drug or biological product undergoes rigorous testing in animals and humans for review by the United States Food and Drug Administration (FDA) before it becomes available for human use. A sponsor files an investigational new drug (IND) application with the FDA with supporting animal data, after which testing is continued with humans. There is sufficient guidance from the FDA on new small molecules and biological products as to which preclinical studies are to be conducted. Synthetic peptides present a unique scenario in which a case-by-case approach is needed for the conduct of preclinical studies. For peptides containing components that are already tested for genotoxicity, it is unnecessary to reevaluate them. If such information can be shared with sponsors ahead of the IND application, it can save time and money. There is no research that evaluated the FDA’s views on pre-IND consultation. Therefore, through a 10-item questionnaire, FDA reviewers’ perceptions on pre-IND consultation, particularly synthetic peptides, were examined. Only four CDER FDA reviewers responded. Three reviewers stated willingness to provide advice to the sponsor through pre-IND discussion and to consult supervisory project managers. Two reviewers agreed that synthetic peptides are to be considered individually for genotoxicity purposes and that pre-testing consultation should be sought. Future research from a larger sample may provide insights on the perceptions of the FDA. Information gleaned from previously approved peptides, however, indicates that there is a wide variability in the type of pre-clinical studies submitted with an NDA (New Drug Application) before progressing to first-in-human studies. However, the routinely submitted studies were single and repeat dose toxicity, genotoxicity, carcinogenicity, reproductive and developmental toxicity, and often studies for impurities.
Niraghatam, Vamsi Vardhan, "Regulatory issues concerning the preclinical testing of synthetic peptides" (2018). Master's Theses and Doctoral Dissertations. 932.