FDA approves fecal microbiota biotherapeutic for recurrent C. difficile infection
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The FDA has approved Rebyota, a microbiota-based live biotherapeutic indicated for the prevention of recurrent Clostridioides difficile infection in adults after antibiotic treatment, Ferring Pharmaceuticals announced in a press release.
The approval comes after an FDA panel voted in favor of Rebyota (fecal microbiota, live-jslm; Ferring Pharmaceuticals) in September 2022, with the Vaccines and Related Biological Products Advisory Committee voting 13-4 in favor of the availability of adequate data supporting effectiveness and 12-4, with one abstention, in favor of the availability of adequate data supporting safety.
“When considering the management of C. difficile infection, the better part of the last decade has been spent chasing the dream of a codified form of microbiota restoration therapy that both patients and providers can expect predictable effectiveness and safety,” Paul Feuerstadt, MD, FACG, AGAF, of Yale University School of Medicine, told Healio. “That day has come, and I could not be more delighted for our medical community.”
According to the release, the FDA approval is based on results from the randomized, double-blind, placebo-controlled phase 3 PUNCH CD3 trial, which demonstrated that a single dose of Rebyota was superior to placebo in reducing recurrence of C. difficile infection (CDI) after standard-of-care antibiotic treatment. The trial included 262 patients, of whom 177 received Rebyota and 85 received placebo.
Treatment success, defined as the absence of CDI diarrhea within 8 weeks after treatment completion, was reported in 70.6% of patients receiving Rebyota compared with 57.5% in the placebo group, the release stated. More than 90% of patients who achieved treatment success were CDI recurrence-free through 6 months.
According to Ferring, study-reported adverse events related to Rebyota were mild to moderate in severity and included gastrointestinal symptoms. There were no serious treatment-emergent adverse events.
“We believe this is a major breakthrough in harnessing the power of the human microbiome to address significant unmet medical needs,” Per Falk, president of Ferring Pharmaceuticals, said in the release. “This is the first FDA approval of a live biotherapeutic and the culmination of decades of research and clinical development. 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:
- FDA panel votes in favor of fecal transplant therapy for recurrent C. difficile infection. https://www.healio.com/news/gastroenterology/20220928/fda-panel-votes-in-favor-of-fecal-transplant-therapy-for-recurrent-c-difficile-infection. Published Sept. 28, 2022. Accessed Dec. 1, 2022.
Perspective
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We are entering an exciting period of restorative microbiome therapeutics. Of the nearly 500,000 patients who develop Clostridioides difficile infection (CDI) every year in the United States, 25% will enter a cycle of recurrent disease with each recurrence increasing the risk for another. This can have a huge impact on a patient’s life.
Recurrent CDI is thought to occur because antibiotics successfully treat the current CDI but do not treat C. difficile spores, which leads to further infection when the antibiotics are stopped. Over the past decade, fecal microbiota transplantation (FMT) has been shown in many controlled studies to be effective in breaking the cycle of recurrent CDI. It appears to act by restoring the gut microbiota, which has been shown to inhibit spore germination.
With the FDA approval of Rebyota, we have the first commercially available FMT product that has passed rigorous FDA requirements for phase 1, 2 and 3 trials.
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