On Wednesday, the Food and Drug Administration approved the first treatment that uses bacteria taken from stool samples to treat intestinal diseases, paving the way for several similar treatments.
A drug called Rebyota is given as a single-dose treatment for C. diff. infections, where bacteria-producing toxins disrupt the normal balance of the human digestive system. Ryota is designed to restore balance by introducing good bacteria taken from donor stool samples.
“We are pleased that the FDA has approved the first fecal microbiome restoration treatment for recurrent C. diff.” “This is a big step forward for the nearly 200,000 people who fight CDI every year,” said Christian John Lillis, executive director of the C. diff charity Peggy Lillis Foundation.
The advisory committee set the stage for approval in September when it voted.
The data showed that Rebyota effectively reduced the recurrence of C. diff. Several outside experts called in to advise the FDA said bringing a regulated product to market would help because similar do-it-yourself methods, called fecal microbiota transplantation (FMT), have been administered over the counter for years.
“Now, remember what’s available to patients: that they get FMT from their roommate, or if it’s available, they get some FMT that’s gone through some kind of quality control,” said Eric Rubin, a member of the advisory committee in a meeting.
In a phase 3 study, the drug reduced C. diff flares by 29%. Ferring, which announced the best results for 2020, published full details of this study earlier this month.
The drug was originally developed by the Minnesota company Rebiotix Inc., which Ferring acquired in 2018. Financial terms were not disclosed.
Rebiotix has been in development for about ten years; the pivotal phase 1 study was first published on ClinicalTrials.gov in 2013.
This decade has not exactly been a smooth one for microbiome therapy. While investors were interested in bacteria-based drugs in the early 2010s, interest declined sharply in 2016 after a major clinical trial failed.
But the field is back, and patient advocates, drug developers, and even regulators have been waiting for this day for years.
In 2019, the FDA held a meeting focused on the future of fecal transplants. Much of the testimony revolved around the perceived and potential competition of such implants and products developed with FDA approval in mind.
FMT has been lightly regulated by the FDA for years; it previously ruled that it has the authority to regulate those transplants, but it will prevent them as long as doctors follow certain guidelines.