FDA Approves First-in-Class Microbiota-Based Live Biotherapeutic for C. Difficile Infection

The FDA has approved fecal microbiota, live-jslm (Rebyota; Ferring Pharmaceuticals) for the prevention of recurring Clostridioides difficile infection (CDI) among patients 18 years of age and older after an antibiotic treatment for recurrent CDI. 

The novel, first-in-class, microbiota-based live biotherapeutic comes in a pre-packaged, single-dose, 150 mL microbiota suspension for rectal administration. Rebyota is sourced from qualified donors and tested for a panel of transmissible pathogens.

“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,” said Paul Feuerstadt, MD, FACG, AGAF, Yale University School of Medicine, in a press release.

The FDA granted approval to Rebyota based on findings from a clinical program that included results from the randomized, double-blind, placebo-controlled, phase 3 PUNCH CD3 trial. The data show that a single dose of Rebyota demonstrated superiority compared with placebo in decreasing the recurrence of CDI following standard-of-care antibiotic treatment.

In the trial, 262 participants were administered blinded treatment (n=177, Rebyota; n=85, placebo) with a primary endpoint of treatment success defined as the absence of CDI diarrhea within 8 weeks following completion of the study treatment.

The Bayesian model-estimated treatment success rate at 8 weeks for Rebyota was 70.6% compared with 57.5% for placebo. The study showed a 99.1% posterior probability that Rebyota was superior to placebo in reducing recurrent CDI following standard-of-care antibiotic treatment. Additionally, more than 90% of participants who achieved treatment success remained free of CDI recurrence through 6 months.

“Rebyota is a much-needed new treatment that offers hope to the thousands of people who suffer from recurrent C. difficile infection each year,” said Brent Ragans, president, Ferring Pharmaceuticals US, in a press release. “Rebyota has the potential to impact not only patients and caregivers, but also the health care system.”

Adverse events (AEs) in the trial were primarily mild-to-moderate with no treatment-related serious AEs. The incidence of treatment-emergent AEs was higher in patients administered Rebyota vs placebo (55.6%, n=100/180, Rebyota; 44.8%, n=39/87, placebo). These AEs were mostly driven by a higher incidence of mild gastrointestinal events, according to the study.

“We believe this is a major breakthrough in harnessing the power of the human microbiome to address significant unmet medical needs. This is the first FDA approval of a live biotherapeutic and the culmination of decades of research and clinical development,” said Per Falk, president, Ferring Pharmaceuticals. “Today’s announcement is not just a milestone for people living with recurrent C. difficile infection, but also represents a significant step which holds promise that many other diseases might be better understood, diagnosed, prevented and treated using our rapidly evolving insights on the role of the microbiome in human health and disease.”

Reference

Ferring Receives U.S. FDA Approval for REBYOTA® (fecal microbiota, live-jslm) – A Novel First-in-Class Microbiota-Based Live Biotherapeutic. Ferring Pharmaceuticals. News release. November 30, 2022. https://ferringusa.com/?press=ferring-receives-u-s-fda-approval-for-rebyota-fecal-microbiota-live-jslm-a-novel-first-in-class-microbiota-based-live-biotherapeutic