FDA Commissioner Marty Makary called these changes “common-sense reforms” that could expedite the development of cell and gene therapies.
The FDA is taking a more flexible approach to regulating cell and gene therapies, easing some of its chemistry, manufacturing and control requirements for these biologics applications.
In a pair of news releases on Sunday, the agency outlined what Commissioner Marty Makary called “common-sense reforms” that take into account the complexity of cell and gene therapies, which are often individualized to a patient or are manufactured in small batches. The Center for Biologics Evaluation and Research has made some concessions on certain requirements “that accommodate the unique characteristics of these innovative therapies.”
For instance, as an investigational gene therapy moves beyond Phase I, the FDA will not require a manufacturer to comply with certain manufacturing specifications for biologics, as outlined in Chapter 600 of the Code of Federal Regulations. Such manufacturing standards otherwise apply to all drug candidates that enter Phase II or Phase III, as per the same Code.
As a cellular or genetic medicine moves into later stages of development, the FDA will also be lenient regarding “minor manufacturing changes,” providing that a drugmaker is able to show that such alterations in production do not meaningfully change the product.
Aside from these manufacturing concessions, the FDA also noted that it would be open to alterations to a product’s commercial specifications.
Despite these regulatory concessions, the agency stated that its regulatory process for cell and gene therapies will maintain “rigorous quality standards through appropriate control measures.” The new flexibilities, it added, “will enable progress while not compromising or undermining the FDA’s ability to assure safety, purity and potency of a product.”
The FDA in recent months has enacted a series of policy changes geared toward assisting developers of cell and gene therapies. In November last year, for instance, the agency rolled out its plausible mechanism pathway, specifically designed to facilitate the development of therapies for diseases affecting small numbers of patients—many of which are CGTs. Under this scheme, the regulator will take into account the biological underpinnings of a disease and how the investigational therapy addresses it.
A few weeks earlier, in October 2025, the FDA released several draft guidance documents for streamlining the review of cell and gene therapies. Among the changes outlined in these documents is the use of self-controlled trials, which will allow developers to use a patient’s own baseline characteristics as a reference point to assess a therapy’s effectiveness.
