FDA Approves First Fecal Microbiota Product

The U.S. Food and Drug Administration on November 30 approved RBX2660 (fecal microbiota, live-jslm, trade name Rebyota) for the prevention of recurrent Clostridioides difficile infection (CDI) in individuals 18 years of age and older.

The novel first-in-class, microbiota-based live biotherapeutic is approved for use after a patient has completed antibiotic treatment for recurrent CDI. FDA granted the application Fast Track, Breakthrough Therapy, and Orphan designations. It is administered rectally as a single dose.

CDI is a potentially life-threatening disease that causes diarrhea and significant inflammation of the colon. In the United States, CDI is associated with 15,000-30,000 deaths annually. People with inflammatory bowel disease (IBD) are particularly at risk of developing CDI.

The new treatment is manufactured from stool donated by qualified persons and tested for a panel of transmissible pathogens. However, FDA cautions that because the treatment is “manufactured from human fecal matter, it may carry a risk of transmitting infectious agents. In addition, Rebyota may contain food allergens; the potential for the product to cause adverse reactions due to food allergens is unknown.”

Two randomized, double-blind, placebo-controlled clinical studies and 1 open-label clinical studies conducted in the United States and in Canada provided the safety data, while  effectiveness was evaluated in an analysis of data from a randomized, double-blind, placebo-controlled, multicenter study. Success in preventing recurrent CDI was defined as the absence of CDI diarrhea within 8 weeks of administration. Statistical analysis estimated success in preventing recurrent CDI through 8 weeks as 70.6% in the treatment group compared to 57.5% in the placebo group.

Rebyota is not indicated for the first occurrence of C. diff, FDA noted. The most common adverse effects among trial participantes were stomach pain (8.9%), diarrhea (7.2%), bloating (3.9%), gas (3.3%), and nausea (3.3%). The studies conducted did not include patients under the age of 18 and did not determine if patients over 65 years of age responded differently to treatment than younger adults.

The FDA granted approval of Rebyota to Ferring Pharmaceuticals Inc.

—Rebecca Mashaw

References:

FDA approves first fecal microbiota product. News release. US Food and Drug Administration; November 30, 2022. Accessed December 1, 2022. https://www.fda.gov/news-events/press-announcements/fda-approves-first-fecal-microbiota-product

Ferring receives U.S. FDA approval for REBYOTA™ (fecal microbiota, live-jslm) – A novel first-in-class microbiota-based live biotherapeutic. News release; December 1, 2022. Accessed December 1, 2022. https://www.ferring.com/ferring-receives-u-s-fda-approval-for-rebyota-fecal-microbiota-live-jslm-a-novel-first-in-class-microbiota-based-live-biotherapeutic/