By Philip W. Wills, Chief Commercial Officer, Catalent Paragon Gene Therapy
Originally published in The Medicine Maker. Copyright 2020 Texere Publishing Limited. All rights reserved.
Since the first market approvals of gene therapy products in 2017, growth in the sector has accelerated. There is now an abundance of gene therapy-related activity, a clinical pipeline in a high growth phase, and an influx of venture capital funding for gene therapy companies. Frost & Sullivan recently reported that, “There are more than 400 cell and gene therapies in preclinical to phase 3 development” (1). In 2018, there was a 27 percent year-on-year increase in the number of clinical trials involving gene therapies, and the FDA’s Commissioner of Food and Drugs predicted that by 2020 the agency will be receiving more than 200 investigational new drug applications per year, and approving 10 to 20 cell and gene therapy products every year by 2025 (2). The regulatory landscape for gene therapy products is not all that different from standard biologics, with the exception that they mostly target rare, orphan diseases. The FDA has proven to be very collaborative for products with this focus, and often designates products for priority review and accelerated approval because of unmet needs.
Gene therapy is a rapidly growing area in healthcare but, now the promise has been shown, we must focus on improving the manufacturing process and reducing costs.