Ferring’s Rebyota, a novel first-in-class microbiota-based live biotherapeutic, has been approved by the FDA for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years and older, following antibiotic treatment for recurrent CDI.
Ferring’s Rebyota (fecal microbiota, live-jslm) was approved by the FDA yesterday for the prevention of recurrence of Clostridioides difficile infection (CDI) in individuals 18 years and older, following antibiotic treatment for recurrent CDI.
Previously called RBX2660, the approval of the novel first-in-class microbiota-based live biotherapeutic was based on the results of the clinical program including the randomized, double-blind, placebo-controlled phase 3 PUNCHCD3 trial.
Findings showed that a single dose of Rebyota was safe and superior to placebo in significantly reducing recurrence of CDI after standard-of-care antibiotic treatment over 8 weeks, with a sustained response achieved through 6 months.
Following these findings, an FDA advisory committee issued a positive vote for its potential in reducing recurrent CDI after antibiotic treatment among adults 18 years and older.
“Until now, patients living with the devastating cycle of recurrent CDI 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, gastroenterologist and assistant clinical professor of medicine, Yale University School of Medicine, in a press release.
CDI is the most common health care–associated infection in US adults, causing symptoms such as severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea, and colitis, which can prove fatal.
An estimated 170,000 cases of CDI occur per year, in which more than 1 in 5 cases recur after initial diagnosis. And after the first recurrence, up to 65% of patients may develop a subsequent recurrence.
Patients with recurrent CDI are at signficantly increased risk for worse outcomes and greater health care resource utilization. A recent study showed that approximately half of all CDI costs are attributable to recurrent CDI ($2.8 billion annually).
Antibiotics currently serve as the established standard-of-care for treatment of CDI, but fecal microbiota transplants, classified by the regulator as investigational, have long been used in the United States for the treatment of the infection.
Antibiotics can also potentially serve as a contributing factor for recurrence, with recently updated clinical practice guidelines recommending fidaxomicin for antibiotic use in initial or recurrent CDI episodes.
“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 Ferring’s president Per Falk, MD, PhD, in a press release.
“Today’s announcement is not just a milestone for people living with recurrent CDI, 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.”