The U.S. Food and Drug Administration expects product approvals for cell and gene therapies to grow in the coming years owing to a large increase in investigational new drug applications.
By the year 2020, the U.S. regulator expects to receive more than 200 of the applications, called INDs, annually, which add to more than 800 active cell-based or directly administered gene therapy INDs now on file. A Jan. 15 statement from Commissioner Scott Gottlieb and Peter Marks, director of the agency's Center for Biologics Evaluation and Research, said the FDA will add about 50 additional clinical reviewers to bolster its group, which will help the agency approve 10 to 20 cell and gene therapy products a year by 2025.
Cell therapies transfer live cells, either a patient's own or from a donor, to tackle diseases such as cancer. Gene therapies introduce, remove or change a person's genetic material to treat or cure a disease, usually through a carrier or vector. The therapies are also referred to as regenerative medicine. Gottlieb said they have the potential to cure rare diseases that have long stymied medical researchers.
The FDA attributed the surge in INDs to a rise in cell and gene therapy products entering early development. Based on this activity, the agency plans to issue new guidance and policies in 2019, which it previewed in the statement.
First, the agency will work with sponsors to find the best use for its expedited programs including accelerated approvals and regenerative medicine advanced therapy designations. The U.S. regulator believes that the accelerated approval pathway offers new opportunities for gene therapy products offering a meaningful therapeutic advantage over available therapies for a serious or life-threatening disease or condition.
Drug developers will be able to use the new guidance documents to introduce advances in manufacturing that promote more efficient development and application of cellular therapy products without necessarily requiring costly new clinical investigations. The guidance will introduce advances for chimeric antigen receptor, or CAR-T, cellular therapies, which take a person's own immune cells and modify them to fight cancer cells when infused back into the body.
The agency plans to hold public meetings with stakeholders to discuss ways to expedite the necessary clinical bridging studies related to development and manufacturing processes. Moreover, the FDA will introduce a trial design for researchers to pool their clinical data, which will help develop a more robust data set for the purpose of getting approval for their biologic products. The agency said through this design it aims to help small sponsors, such as academic investigators, who may not have sufficient sources to conduct a study.
Gottlieb and Marks' statement comes amid a federal government shutdown, the longest in U.S. history. Gottlieb has said his agency is unable to accept any new user fees or any applications that require a fee for new drug applications while the shutdown continues.