The guidance is intended to provide sponsors who are developing cellular therapies for the treatment of cardiac disease, with recommendations on the design of preclinical and clinical studies, and on the chemistry, manufacturing, and controls (CMC) information to include in an investigational new drug application (IND) for cardiac cellular therapy.
This guidance provides to sponsors developing cellular therapies for the treatment of cardiac disease with recommendations including, but not limited to, the following: (1) Design of preclinical and clinical studies; (2) information to submit on the product delivery system; and (3) the chemistry, manufacturing and controls information to include in an IND for cardiac cellular therapy. This guidance also includes regulatory considerations for the use of intravascular catheter delivery systems including recommendations regarding the information that sponsors should submit on the product’s delivery system.
The Background section of the guidance states as follows:
There has been much debate about how to interpret differing data and theories on the mechanism of action of cell therapies in cardiovascular disease. Clearly the agency is looking to set some standards in the design of these trials. I'll let colleagues more qualified than I on the subject comment on what they've accomplished with the draft guidance.
Cellular therapies for the treatment of cardiac disease generally meet the definition of “biological product” in section 351(i) of the Public Health Service Act (42 U.S.C. 262(i)), and are regulated by the Center for Biologics Evaluation and Research (CBER). Intravascular catheters and other drug and biologic delivery systems generally meet the definition of “device” under section 201(h) of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 321(h)).
FDA expects that these cellular therapies and their dedicated delivery systems would meet the definition of a combination product under 21 CFR 3.2(e), either by being co-packaged or, if marketed separately, by using labeling that indicates they are intended for use together. Based on available information, the somatic cellular therapy generally would provide the primary mode of action of the combination product and the lead center typically would be CBER in accordance with 21 CFR 3.4.
For a combination product, a single investigational application is generally sufficient. For cellular therapies and their dedicated delivery systems, this generally would be an IND. The IND application should contain the information as described in this guidance on the biological product and on the delivery system, along with any other information as required by the applicable regulations. CBER and CDRH intend to consult or collaborate on the IND review, as appropriate for the science and technology of the combination product.For a combination product, one marketing application is also generally sufficient for FDA review and marketing authorization. However, sometimes two marketing applications may be necessary.