Ferring Inc. has achieved a significant milestone in the field of microbiome-based therapeutics with the approval of REBYOTA® (fecal microbiota, live) by Health Canada. This novel, first-in-class microbiome-restoration therapy is the latest advancement in the battle against Clostridioides difficile (C. diff), a dangerous and often recurring bacterial infection that affects hospitalized patients worldwide.

REBYOTA® is designed to prevent the recurrence of C. diff in individuals aged 18 and older, following standard antibiotic treatment for recurrent infections. However, it is important to note that REBYOTA® is not a treatment for active C. diff infections. This approval marks a groundbreaking moment for patients suffering from the debilitating cycle of recurrent C. diff infections, which can drastically impact both quality of life and the healthcare system.

A vicious cycle: the burden of recurrent c. diff

C. diff is the leading cause of infectious diarrhea in hospitalized patients, especially in industrialized nations like Canada. While antibiotics can address the infection, they often disrupt the gut microbiota, leading to recurrence. Up to 35% of C. diff cases can relapse after initial treatment, with patients who experience one recurrence at higher risk for subsequent infections.

The vicious cycle of C. diff recurrence places a significant burden on patients, caregivers, and healthcare systems alike. “REBYOTA offers a much-needed solution to break this cycle,” says Gail Attara, CEO and Co-Founder of the Gastrointestinal Society. “It provides hope to those suffering from this chronic condition by restoring the microbiome to a healthier state, helping to prevent future infections and reducing hospital admissions.”

Clinical trials: the largest program in microbiome therapy

Health Canada’s approval of REBYOTA® is based on a robust clinical program, which includes five pivotal clinical trials and over 1,000 participants—more than 230 of whom were Canadian patients enrolled in five clinical sites across Canada. The cornerstone of this program was the PUNCH™ CD3 trial, a randomized, double-blind, placebo-controlled Phase 3 study that evaluated the safety and efficacy of REBYOTA® in preventing recurrent C. diff infections.

The results of the Canadian cohort (117 participants) were presented at the Canadian Digestive Disease Week (CDDW) in Quebec City. In this cohort, 75.2% of patients who received REBYOTA® after antibiotic treatment achieved treatment success, and 90.9% sustained clinical response at six months.

With 5 additional studies involving 978 trial participants over six months, REBYOTA® demonstrated strong safety and efficacy, with adverse events primarily being mild to moderate and related to preexisting conditions. The treatment was well tolerated, with a similar incidence of treatment-emergent adverse events (TEAEs) between REBYOTA® and placebo groups.

A game-changer for Canadian healthcare

The approval of REBYOTA® is not only a major achievement in the field of microbiome therapeutics but also a critical development for Canadian healthcare. Dr. Theodore Steiner, Head of Infectious Disease at the University of British Columbia, emphasized the importance of this approval: “Recurrent C. diff is more than just a physical burden—it affects all aspects of a patient’s life. For the first time, we now have an approved therapy to prevent future recurrences, offering much-needed relief to patients who have been trapped in the cycle of recurrent infections.”

By restoring the microbiome and reducing the need for chronic antibiotic treatments, REBYOTA® has the potential to significantly reduce healthcare costs by keeping patients out of the hospital. This aligns with Ferring’s commitment to addressing unmet needs in gastrointestinal health.

The path forward: exploring the microbiome’s potential

This approval represents the culmination of decades of research and development in the field of microbiome restoration. “The results from the PUNCH-CD program, especially the Canadian cohort of the study, represent a breakthrough in utilizing the human microbiome to address medical needs,” said Lee Ferreira, General Manager of Ferring Inc. Canada. “We believe this is just the beginning. As we continue to explore the role of the microbiome in human health, we are confident that microbiome-based therapies will open new possibilities for treating a wide range of conditions.”

Ferring is also working on other microbiome-based therapies and aims to broaden the application of its technologies to address other conditions. As the field of microbiome therapeutics continues to evolve, Ferring is positioning itself at the forefront of innovation, working to help patients live better lives.

Looking to the future: a new era in microbiome therapeutics

The approval of REBYOTA® is an important step in Ferring’s mission to harness the potential of the human microbiome to develop novel therapeutics for a range of gastrointestinal and other diseases. As the first approved therapy of its kind for recurrent C. diff, REBYOTA® opens up new possibilities for the microbiome’s role in healthcare and highlights the increasing importance of microbiome research in the development of future treatments. For patients, caregivers, and healthcare providers, this approval provides a promising new option to combat the ongoing challenge of recurrent C. diff infections. With its potential to significantly improve patient outcomes and reduce healthcare costs, REBYOTA® stands as a testament to the power of innovation in microbiome-based medicine.