Cell and gene therapies (CGTs) represent the next great wave of therapeutic innovation and have led to the development of promising therapies worldwide. While a few of the initial therapies have received marketing approval in the United States (U.S.), there are a number of promising therapies in development outside of the U.S. As these therapies advance through preclinical and clinical development, many companies are considering if, when, and how they should bring their therapies to the U.S. to unlock the commercial potential of the world’s largest biopharmaceutical market.
The unique aspects of each therapy, along with the rapidly changing regulatory environment, have led to a variety of development options. Sponsors considering bringing CGTs to the U.S. at various points during preclinical and clinical development must take into consideration the variety of potential implications to their development plan, including impact on development cost, timeline, feasibility and ability to leverage existing data.
This whitepaper summarizes the process and considerations for bringing a preclinical or clinical state CGT to the U.S. for development towards a successful submission of an Investigational New Drug Application (IND). By understanding the implications of each of the core components of an IND submission. CGT companies will be prepared to navigate the potential complexities of the U.S. regulatory and drug development landscape.
Leveraging a collaborative regulatory process
Before bringing investigational therapies to the U.S., ex-U.S. CGT companies should first determine how to navigate the U.S. regulatory approval process and successfully submit an IND. The U.S. Food and Drug Administration (FDA) has established a collaborative regulatory process for CGTs with early and regular engagement with the Sponsor, as well as special regulatory designations useful for many CGTs. Specifically, to support the development of CGT products to reach regulatory milestones in the U.S., the FDA offers increased interaction and collaboration with the Sponsor leading up to the submission of an IND while the special regulatory designations described later in the paper can allow for more frequent interactions with the FDA during the clinical phase of development.