Providing an overview of the submissions process and examples of U.S. Food and Drug Administration (FDA) applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), a new article can help developers of gene therapies for rare genetic diseases.

Anne Pariser and Elizabeth Ottinger, from the National Center for Advancing in Translational Sciences (NCATS), National Institutes of Health, and coauthors, describe the ODD and RPDD programs, which provide financial incentives for the development of diagnostic drugs, preventive measures, and treatments of diseases affecting small patient populations.

Orphan drug designation (ODD) is an important program intended to facilitate the development of orphan drugs in the United States. An orphan drug benefiting pediatric patients can qualify as a drug for a Rare Pediatric Disease Designation (RPDD) as well. The ODD and RPDD programs provide financial incentives for development of diagnostic drugs, preventive measures, and treatment of diseases affecting small patient populations (adult and pediatric) for which commercial development would otherwise be very challenging.

The ODD application compilation process

The application can be compiled as per the FDA requirements at different program stages once the lead investigational drug product has been identified. The application must include proof of direct benefit, documentation of population size, and orphan drug regulatory history. Direct benefit information and population size are critical, and substantial details in these sections make a strong application. FDA, Food and Drug Administration; ODD, orphan drug designation.

In 2019, a multidisciplinary group of collaborators at National Institutes of Health (NIH) embarked upon a gene therapy platform program called Platform Vector Gene Therapy (PaVe-GT) intended to develop gene therapies for four such rare disorders. An important part of PaVe-GT is to publicly share scientific and regulatory experience gained at different stages during the implementation of the PaVe-GT platform utilizing illustrative examples. The PaVe-GT team recently obtained ODD and RPDD for an adeno-associated virus gene therapy to treat propionic acidemia.

Given an increasing interest in obtaining ODD for gene therapy, especially by small companies, research investigators, and patient groups, the authors overview the submission process and subsequently provide examples of our ODD and RPDD applications. These ODD and RPDD applications and templates can also be found on the PaVe-GT website. Shared reference documents will have great utility to assist parties who may have limited experience with the preparation of similar applications for their orphan product.

“The PaVe-GT program is pioneering regulatory and clinical trial approaches to broaden the impact of gene therapy,” says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Chan Medical School.

In this article, members of the PaVe-GT program emphasize the significance of these incentive programs in stimulating drug development and illustrate how developers of gene therapies can utilize FDA guidance to prepare ODD or RPDD applications.

Source – Mary Ann Liebert, Inc., Publishers

Lomash RM, Shchelochkov O, Chandler RJ, Venditti CP, Pariser AR, Ottinger EA, NIH PaVe-GT Team. (2023) Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Experience. Hum Gene Ther 34(5-6):217-227. doi: 10.1089/hum.2022.232

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