Ferring Receives U.S. FDA Approval for REBYOTA® (fecal microbiota, live-jslm) ? A Novel First-in-Class Microbiota-Based Live Biotherapeutic

Ferring Pharmaceuticals today announced the U.S. Food and Drug Administration (FDA) approved 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.

"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, M.D., F.A.C.G., A.G.A.F., Yale University School of Medicine.

"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 PUNCHtm 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. Two hundred sixty-two (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.¹ More than 90% of study participants who achieved treatment success remained free of CDI recurrence through six months.²

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.²

"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."

Please see important safety information below, and for full prescribing information visit: https://www.ferringusa.com/pi/rebyota.

About C. difficile Infection (CDI)

CDI is a serious and potentially deadly infection that impacts people across the globe. The C. difficile bacterium causes debilitating symptoms, such as severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea and colitis (an inflammation of the colon).³ CDI can be the start of a vicious cycle of recurrence, causing a significant burden for patients and the healthcare system.^4,5 It has been estimated that up to 35% of CDI cases recur after initial diagnosis and people who have had a recurrence are at significantly higher risk of further infections.^6,7,8,9 After the first recurrence, it has been estimated that up to 65% of patients may develop a subsequent recurrence.^8,9 Antibiotics ? the current standard of care for treatment of CDI ? treat the disease but can also be a contributing factor for a vicious cycle of recurrence, causing a significant burden for patients suffering from this debilitating and potentially deadly illness.^3,10

ABOUT REBYOTA

REBYOTA is 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 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.

INDICATION

REBYOTA is indicated for the prevention of recurrence of Clostridioides difficile (C. diff) infection in individuals 18 years of age and older, following antibiotic treatment for recurrent C. diff.

Limitation of Use:

REBYOTA is not indicated for the first occurrence of C. diff.

IMPORTANT SAFETY INFORMATION

• You should not receive REBYOTA if you have a history of a severe allergic reaction (e.g. anaphylaxis) to REBYOTA or any of its components.
• You should report to your doctor any infection you think you may have acquired after administration.
• Prior to treatment with REBYOTA, talk to your doctor about the possibility of a sudden allergic reaction following administration.
• Talk to your doctor about any known food allergies.
• Most common side effects may include stomach pain (8.9%), diarrhea (7.2%), bloating (3.9%), gas (3.3%), and nausea (3.3%).
• REBYOTA has not been studied in patients below 18 years of age.
• Clinical studies did not determine if adults 65 years of age and older responded differently than younger adults.

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088.

Please click to see the full Prescribing Information.

About Ferring Pharmaceuticals

Ferring Pharmaceuticals is a research-driven, specialty biopharmaceutical group committed to helping people around the world build families and live better lives. Headquartered in Saint-Prex, Switzerland, Ferring is a leader in reproductive medicine and women's health, and in specialty areas within gastroenterology and urology. Ferring has been developing treatments for mothers and babies for over 50 years and has a portfolio covering treatments from conception to birth. Founded in 1950, privately owned Ferring now employs around 6,000 people worldwide, has its own operating subsidiaries in more than 50 countries, and markets its products in 110 countries.

Learn more at www.ferring.com, or connect with us on Twitter, Facebook, Instagram, LinkedIn and YouTube.

Ferring is committed to exploring the crucial link between the microbiome and human health, beginning with the threat of recurrent C. difficile infection. Ferring is working to develop novel microbiome-based therapeutics to address significant unmet needs and help people live better lives. Connect with us on our dedicated microbiome therapeutics development channels on Twitter and LinkedIn.

References:

1. REBYOTA. Prescribing information. Parsippany, NJ: Ferring Pharmaceuticals Inc; 2022.
2. Khanna, S, Assi, M, Lee, C, et al. Efficacy and safety of RBX2660 in PUNCH CD3, a Phase III, randomized, double-blind, placebo-controlled trial with a Bayesian primary analysis for the prevention of recurrent Clostridioides difficile infection. Drugs (2022). Available at: https://doi.org/10.1007/s40265-022-01797-x.
3. Centers for Disease Control and Prevention. What Is C. Diff? 17 Dec. 2018. Available at: https://www.cdc.gov/cdiff/what-is.html.
4. Centers for Disease Control and Prevention. 24 June 2020. Available at: https://www.cdc.gov/drugresistance/pdf/threats-report/clostridioides-difficile-508.pdf.
5. Feuerstadt P, et al. J Med Econ. 2020;23(6):603-609.
6. Riddle DJ, Dubberke ER. Clostridium difficile infection in the intensive care unit. Infect Dis Clin North Am. 2009;23(3):727-743.
7. Nelson WW, et al. Health care resource utilization and costs of recurrent Clostridioides difficile infection in the elderly: a real-world claims analysis. J Manag Care Spec Pharm. 2021;27(7):828-838. doi: 10.18553/jmcp.2021.20395. Epub 2021 Mar 11.
8. Kelly, CP. Can we identify patients at high risk of recurrent Clostridium difficile infection? Clin Microbiol Infect. 2012;18(suppl 6):21?27.
9. Smits WK, et al. Clostridium difficile infection. Nat Rev Dis Primers. 2016;2:16020. doi: 10.1038/nrdp.2016.20.
10. Lessa FC, Mu Y, Bamberg WM, et al. Burden of Clostridium difficile infection in the United States. N Engl J Med. 2015;372(9):825-834.