June 13, 2019
“The medical community is actively engaged in exploring the potential uses of Fecal Microbiota for Transplantation, or FMT. Although FMT is not approved by the FDA for any use, the agency plays a critical role in supporting product development while assessing the risks and benefits to patients of unapproved therapies. The agency also works to strike a balance between assuring patient safety and facilitating access to unapproved treatments for unmet medical needs. For example, the FDA has issued guidance stating that it intends to exercise enforcement discretion, under limited conditions, regarding the investigational new drug requirements for FMT used to treat Clostridium difficile infection unresponsive to standard therapies,” said Peter Marks, M.D., Ph.D., director of FDA’s Center for Biologics Evaluation and Research. “While we support this area of scientific discovery, it’s important to note that FMT does not come without risk. We’ve become aware of infections with multi-drug resistant organisms after patients received investigational FMT, including one patient death. We therefore want to alert all health care professionals who administer FMT about this potential serious risk so they can inform their patients. Today’s safety communication underscores the importance of why new therapies are thoroughly studied to ensure the benefits of taking them outweigh the risks to patients, and we will continue to aggressively monitor clinical trials to ensure patients are protected when safety concerns arise.”
Today, the U.S. Food and Drug Administration issued a safety communication regarding the use of Fecal Microbiota for Transplantation (FMT) and the risk of serious or life-threatening infections due to transmission of multi-drug resistant organisms (MDROs), as well as protections FDA has determined are needed for patients receiving investigational FMT.
Two immunocompromised adults who received investigational FMT developed invasive bacterial infections due to the transmission of a MDRO from FMT; one of the patients died. Because of these serious adverse reactions, FDA has determined that certain donor screening and stool testing protections are needed for any investigational use of FMT, as outlined in the safety communication.
Additionally, today’s safety communication alerts health care professionals about the potential risk of MDRO transmission by FMT and resultant serious adverse outcomes that may occur. It is also a reminder that, as described in the FDA’s guidance, the agency intends to exercise enforcement discretion regarding the IND requirements for the use of FMT to treat C. difficile infections in patients who have not responded to standard therapies provided that the treating physician obtains adequate informed consent, which should include a statement that the use of FMT to treat C. difficile is investigational and a discussion of potential risks. The FDA encourages health care professionals and patients to report any adverse events or side effects to the FDA’s MedWatch Adverse Event Reporting program.
- Important Safety Alert Regarding Use of Fecal Microbiota for Transplantation and Risk of Serious Adverse Reactions Due to Transmission of Multi-Drug Resistant Organisms
- FDA Guidance for Industry: Enforcement Policy Regarding Investigational New Drug Requirements for Use of Fecal Microbiota for Transplantation to Treat Clostridium difficile Infection Not Responsive to Standard Therapies
- Information Pertaining to Additional Safety Protections Regarding Use of Fecal Microbiota for Transplantation – Screening and Testing of Stool Donors for Multi-drug Resistant Organisms
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.