The U.S. Food and Drug Administration (FDA) has approved Ferring Pharmaceuticals’ REBYOTA (fecal microbiota, live-jslm), a novel first-in-class microbiota-based live biotherapeutic indicated for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for recurrent CDI.
“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 U.S. “REBYOTA has the potential to impact not only patients and caregivers, but also the healthcare system.”
The FDA approval of REBYOTA is based on the results from the clinical program including the randomized, double-blind, placebo-controlled Phase 3 PUNCH CD3 trial in which a single dose of REBYOTA demonstrated superiority to placebo as a treatment to reduce recurrence of CDI after standard-of-care antibiotic treatment.
262 trial participants received blinded treatment (n=177, REBYOTA; n=85, placebo) and the primary endpoint was treatment success, defined as the absence of CDI diarrhea within eight weeks after completing study treatment.
The Bayesian model-estimated treatment success rate at eight weeks for REBYOTA was 70.6% versus 57.5% for placebo, with a 99.1% posterior probability that REBYOTA was superior to placebo in reducing recurrent CDI after standard-of-care antibiotic treatment. More than 90% of study participants who achieved treatment success remained free of CDI recurrence through six months.
In the study, adverse events (AEs) were primarily mild-to-moderate and there were no treatment-related serious adverse events (SAEs). Incidence of treatment-emergent adverse events (TEAEs) was higher in REBYOTA recipients compared with placebo (55.6%, n=100/180, REBYOTA; 44.8%, n=39/87, placebo), mostly driven by a higher incidence of mild gastrointestinal events.
“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.”