On Nov. 30, FDA approved the first fecal microbiota therapy for recurrent Clostridioides difficile infection (CDI). This approval came days after FDA issued guidance on regulatory requirements for stool banks providing fecal microbiota transplantation (FMT) products. Here is a summary of the regulatory actions and what they mean for GIs and our patients with recurrent CDI.
FDA approves first FMT therapy
FDA approved Rebyota® (Ferring Pharmaceuticals Inc.) for the prevention of recurrence of CDI in patients 18 years of age and older who have completed antibiotic treatment for recurrent CDI.
“This is a major milestone in the translation of gut microbiome science to clinical solutions for patients. This accomplishment is based on decades of work on the gut microbiome by gastroenterologists and collaborators,” says Phillip I. Tarr, MD, chair of the AGA Center for Gut Microbiome Research and Education Scientific Advisory Board. “AGA applauds FDA for recognizing the demonstrated and conceptual merit of microbiota-based therapies.”
FDA requires stool banks to comply with investigational new drug (IND) requirements
In a change of previous guidance, FDA will now require stool banks who provide FMT products to comply with IND requirements, effective immediately. For now, health care professionals can still use appropriately screened material donated by individuals (donor stool) for FMT procedures in patients with recurrent CDI whose symptoms do not respond to antibiotic therapies.
The nonprofit stool bank OpenBiome announced it will continue operating normally under IND and remains committed to providing safe and continued access to FMT. OpenBiome and its partner, the University of Minnesota Microbiota Therapeutics Program, are working with FDA to ensure that their regulatory filings are consistent with the finalized guidance. Related: OpenBiome FAQ.
What does this mean for gastroenterologists, other health care professionals who treat CDI, and patients?
- There is now an FDA-approved, microbiota-based therapy for the prevention of recurrent CDI in adult patients who have previously completed antibiotic treatment.
- Stool banks can continue to operate and provide FMT material as long as they are compliant with the FDA’s IND requirements.
- Health care professionals using FMT material from known donors (i.e., stool provided by individuals and not from a stool bank) should abide by the following requirements outlined in the FDA’s November 2022 final guidance:
- Obtain appropriate informed consent from the patient or their legally authorized representative.
- Perform appropriate screening and testing of the stool donor and stool.
- Follow procedures that mitigate potential safety concerns, such as appropriate storage and handling of donor stool.
AGA looks forward to future advances in this field. We will continue to follow the long-term effectiveness and safety of patients receiving Rebyota, FMT and other microbiota-based therapies through our FMT National Registry. For more information about CDI and FMT, visit patient.gastro.org.
