FDA Committee Recommends Approval for Live Microbiota C Difficile Medication

This article was originally published in ContagionLive.

The US Food and Drug Administration’s (FDA) Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted on September 22 to recommend a Biologics License Application (BLA) for RBX2660 (Rebyota) to treat recurrent Clostridioides difficile infection (CDI).

The treatment, developed by Ferring Pharmaceuticals, is aimed at reducing recurrent CDI after antibiotic usage by being administered as a pre-packaged single-dose, consisting of 150 mL microbiota suspension from individual human stool donation.

VRBPAC voted on the following questions:

• Are the available data adequate to support the effectiveness of REBYOTA to reduce the recurrence of Clostridioides difficile infection (CDI) in adults 18 years of age and older following antibiotic treatment for recurrent CDI?
• Are the available data adequate to support the safety of REBYOTA when administered to adults 18 years of age and older following antibiotic treatment for recurrent CDI?

VRBPAC voted 13 to 4 in favor of the first question, and 12 to 4 in favor of the second, with 1 abstention.

RBX2660 is a standardized, stabilized, investigational microbiota-based live biotherapeutic. It reduces the cycle of recurrent C diff infections by restoring the gut microbiome and inhibiting antibiotic-induced dysbiosis.

There are other treatments for patients with recurrent CDI, such as fecal microbiota transplantation (FMT). However, FMT is only recommended for patients with multiple recurrences who have also failed applicable antibiotic treatments. RBX2660 is intended to treat patients suffering from debilitating CDI, but who may not qualify for FMT.

CDI has a high rate of morbidity and mortality, in addition to draining health care resources. Approximately 500,000 Americans contract C diff each year, and 30,000 die from this highly resistant bacterium. This burden becomes greater and deadlier with each CDI recurrence.

The treatment was previously granted Fast Track, Orphan, and Breakthrough Therapy designations by the FDA. The treatment will be administered as a pre-packaged single-dose, consisting of 150 mL microbiota suspension from individual human stool donation.

Risk factors for CDI include antibiotics, proton pump inhibitors, previous CDI, health care exposure, age above 65 years, and comorbid conditions such as malignancy, kidney disease, inflammatory bowel disease, and immunosuppression.