The Food and Drug Administration approved the first therapy using bacteria from stool samples to treat a bowel disorder on Wednesday, paving the way for several similar treatments in development.
The drug, called Rebyota, is given as a one-dose treatment for clostridium difficile infections, commonly known as C. diff., in which the toxin-producing bacteria disrupts the normal balance in a person’s digestive system. Rebyota is designed to restore balance by introducing good bacteria taken from donor stool samples.
“We are delighted that FDA has approved the first fecal microbiome restoration therapy for recurrent C. diff. This is an enormous step forward for the nearly 200,000 people who battle rCDI each year,” said Christian John Lillis, the executive director of the C. diff advocacy organization the Peggy Lillis Foundation.
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Ferring did not immediately respond to a request for comment.
An advisory committee set the stage for approval in September, when it voted 13 to 4 that the data showed Rebyota was effective in reducing C. diff recurrence. Multiple outside experts brought in to advise the FDA said it would be beneficial to have a regulated product on the market, given that similar DIY treatments called fecal microbiota transplantation (FMT) have been given outside of physicians’ offices for years.
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“Remember right now what’s out there for patients is they can get an FMT from their roommate, or if this is available, they can get a defined FMT that has undergone some sort of quality control,” advisory committee member Eric Rubin said during the meeting.
In a Phase 3 trial, the drug reduced C.diff flare-ups by 29.4%. Ferring, which announced topline results in 2020, published full data from that study earlier this month.
The drug was initially developed by Rebiotix Inc., a Minnesota-based company that Ferring acquired in 2018. The financial terms weren’t disclosed.
Rebyota has been in development for about a decade; a key Phase 1 study was first posted to ClinicalTrials.gov in 2013. That decade hasn’t been entirely smooth sailing for microbiome therapeutics. Although investors were intrigued by bacteria-based drug candidates in the early 2010s, interest dropped sharply in 2016 after a significant clinical trial failed.
But the field did recover — and patient advocates, drug developers and even regulators have been anticipating this day for years.
In 2019, the FDA held a meeting that focused on the future of fecal transplants. Much of the testimony circled around perceived and potential competition between such transplants and products being developed with FDA approval in mind.
For years, the FDA has governed FMTs with a light touch; it previously decided that it had the power to regulate these transplants but would hold off as long as physicians followed specific sets of guidelines.
Noted stool bank OpenBiome announced in 2021 plans to wind down the part of its operation that supplied donor stool to clinicians, though it vowed to continue providing material until the first treatment was approved.
But although the organization did stop producing its own material, said OpenBiome CEO Julie Barrett O’Brien, it established a partnership with the University of Minnesota’s microbiome therapeutics program and has continued to supply FMT treatments.
“While we were winding down our product line, we brought on theirs to make sure we could fill this critical gap,” O’Brien said.
OpenBiome will continue to offer FMTs. “It’s going to take a bit of time to get these products that are approved into the marketplace,” O’Brien said. “We’re going to keep providing these treatments for patients.”
Nevertheless, O’Brien said the approval was “really exciting. We’re thrilled for patients, because honestly — this is such a horrible disease. The more options they have, the better they’ll be.”
Rebiotix is not the only company that has developed a drug for C. diff. Seres Therapeutics announced in September that it had completed its application for FDA approval for its oral microbiome therapeutic intended to treat the condition. The agency is expected to decide on whether to approve the drug by April 26, 2023.
