The FDA has approved the first-ever fecal microbiota product, the agency announced on Wednesday.
Rebyota was approved to prevent the recurrence of Clostridioides difficile (C. difficile) infection in adults who have already completed a course of antibiotics for recurrent C. difficile; it is not indicated for the first occurrence of C. difficile.
The fecal microbiota product is prepared from stool donated by qualified donors, which is tested for transmissible pathogens, and when cleared, prepared for rectal administration via a single dose.
“Today’s approval of Rebyota is an advance in caring for patients who have recurrent C. difficile infection,” Peter Marks, MD, PhD, of FDA’s Center for Biologics Evaluation and Research, said in a statement. “Recurrent [C. difficile infection] impacts an individual’s quality of life and can also potentially be life-threatening. As the first FDA-approved fecal microbiota product, today’s action represents an important milestone, as it provides an additional approved option to prevent recurrent [C. difficile infection].”
C. difficile is a bacteria that can lead to life-threatening infections that cause diarrhea and significant inflammation of the colon. It is associated with 15,000-30,000 deaths each year in the U.S.
“Certain situations, such as taking antibiotics to treat an infection, may change the balance of microorganisms in the gut, allowing C. difficile to multiply and release toxins causing diarrhea, abdominal pain and fever, and in some cases, organ failure and death,” the FDA explained. Factors that increase the risk for infection include older age, hospitalization, a weakened immune system, and a previous history of C. difficile infection.
The fecal microbiota-based product is thought to facilitate restoration of the flora in the gut, preventing inflammation and recurrence of the infection. However, the FDA warns in its announcement that the product is manufactured from human fecal matter, so it could contain food allergens and infectious agents not part of the panel of tested pathogens.
Approval was granted following positive recommendations on the product’s efficacy and safety from the FDA’s Vaccines and Related Biological Products Advisory Committee in September.
Combined data from two randomized placebo-controlled studies involving 344 adults in total showed that treatment success through 8 weeks was significantly higher with the fecal microbiota-based product, with 70.6% avoiding a recurrence of C. difficile diarrhea as compared with 57.5% for those assigned to placebo.
Safety of the product was determined from data on 978 adults with recurrent C. difficile from those two trials plus several open-label studies conducted in the U.S. and Canada. The most common side effects with the product included abdominal pain, diarrhea, abdominal bloating, gas, and nausea, the FDA noted.
In developer Ferring Pharmaceuticals announcement, the company cited safety data from the larger phase III PUNCH CD3 trial showing more treatment-emergent adverse events with the fecal microbiota product compared with placebo (55.6% vs 44.8%). These events were primarily mild-to-moderate, according to the company, and no serious adverse events were deemed related to treatment.