Safety analysis published for C. diff biotherapeutic

Integrated safety analysis from five prospective clinical trials for the first and only single-dose, US Food and Drug Administration (FDA)-approved microbiome-based treatment to prevent recurrent Clostridioides difficile (C. diff) infection after antibiotic treatment, has been published in Therapeutic Advances in Gastroenterology.

The analysis is the largest safety evaluation to date of any microbiota-based live biotherapeutic. Safety data for up to two years is included in the analysis.

Ferring Pharmaceuticals’ REBYOTA^® (faecal microbiota, live – jslm) is indicated for preventing recurrence of C. diff infection in individuals 18 years of age and older.

First faecal microbiota product approved for C. diff

What did the safety analysis show?

Data used in the integrated safety analysis was taken from three Phase II trials evaluating prevention of C. diff recurrence (PUNCH CD, PUNCH CD2, PUNCH Open-Label) and two Phase III trials (PUNCH CD3, PUNCH CD3-OLS) of REBYOTA.

Treatment emergent adverse events (TEAEs) were reported in 60.2 percent of placebo only participants and 66.4 percent of RBL only participants. Only abdominal pain, nausea, and flatulence were significantly higher in the RBL only group compared with the placebo only group. Most TEAEs were mild or moderate in severity. These were most frequently related to pre-existing conditions. No reported infections for which the causative pathogen was traced to RBL were reported. Potentially life-threatening TEAEs were observed in 3.0 percent of participants.

What did data from the five prospective clinical trials for C. diff recurrence reveal?

Of approximately total 1,000 participants included the five trials, 978 participants received at least one dose of REBYOTA (assigned treatment or after C. diff recurrence). There were 83 participants who received placebo only.

The 507 of 763 participants who received multiple doses of REBYOTA or who received placebo and REBYOTA were not included in the analysis and 60.2 percent of placebo-only participants (50 of 83).

“The integrated analysis provides further evidence that REBYOTA is a safe and well-tolerated treatment for patients with recurrent C. diff infection,” commented Dr Paul Feuerstadt, Yale University School of Medicine, and one of the analysis authors.

Microbiome therapies: a maturing movement