FDA Approves Rebyota, the First-ever Microbiota Product

The FDA has approved Rebyota, the first fecal microbiota product for the prevention of recurrence of Clostridioides difficile (C. diff) infection (CDI) in people 18 years of age and older.

The human microbiome is a complex community of microorganisms in and on the body. In the gut, if a microbial imbalance occurs, this can lead to C. diff, irritable bowel syndrome or even diabetes. C. diff is a serious disease that causes severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea, and colitis. It has been estimated that up to 35% of cases recur after initial diagnosis.

Rebyota (formerly RBX2660) is a fecal microbiota transplantation therapy that was developed by Rebiotix, a Ferring Pharmaceuticals company. It is administered rectally as a single dose and is prepared from stool donated by qualified individuals. The donors and the donated stool are tested for a panel of transmissible pathogens.

Paul Feuerstadt, M.D.

“Until now, patients living with the devastating cycle of recurrent C. difficile infection have had limited FDA-approved treatment options, causing them to suffer long periods of time with debilitating symptoms that prevent them from leaving their homes and even separate them from immediate family members,” Paul Feuerstadt, M.D., Yale University School of Medicine, said in a press release.

The approval is based on the results from the clinical program including the phase 3 PUNCH CD3 trial in which a single dose of Rebyota demonstrated superiority to placebo as a treatment to reduce recurrence of C. diff infection after standard-of-care antibiotic treatment. In the trial, 262 people received either Rebyota or placebo, and the primary endpoint was absence of CDI diarrhea within eight weeks after completing study treatment. The success rate at eight weeks for Rebyota was 70.6% versus 57.5% for placebo. More than 90% of study participants who achieved treatment success remained free of C. diff infection recurrence through six months.

In the study, adverse events were primarily mild-to-moderate and there were no treatment-related serious adverse events. Incidence of treatment-emergent adverse events was higher in Rebyota recipients compared with placebo, mostly driven by a higher incidence of mild gastrointestinal events.

Related: FDA Advisory Committee Gives Positive Vote for Microbiota-based C. Diff Therapy

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) issued a positive vote on Sept. 22, 2022, for Rebyota. The committee voted 13 to 4 indicating that the data were adequate to support the effectiveness of RBX2660 to reduce the recurrence of C. difficile infection in adults 18 years of age and older following antibiotic treatment. The committee also voted 12 to 4 with one abstention that the data were adequate to support the safety of RBX2660.