In developing a treatment for Clostridium difficile—an infection that can cause life-threatening diarrhea—Ferring Pharmaceuticals has had to dig deep in a decadelong pursuit, experimenting with bacteria from stool samples.
Wednesday, the agency signed off on Rebyota, a one-time, enema-delivered therapy that can restore proper bacterial balance in the digestive system.
As the first FDA-approved fecal microbiota product, Rebyota represents a significant milestone, potentially clearing the way for other such treatments.
“We believe this is a major breakthrough in harnessing the power of the human microbiome to address significant unmet medical needs,” Per Falk, president of Ferring, said in a release. “(This) presents a significant step which holds promise that many other diseases might be better understood, diagnosed, prevented and treated using our rapidly evolving insights.”
C. diff. is a superbug that resists antibiotics and other medications. The disorder causes roughly 500,000 illnesses and between 15,000 and 30,000 deaths annually in the U.S.
To address C. diff., patients have turned to loosely regulated, investigational fecal microbiota transplants (FMTs), which use healthy bacteria from the feces of donors and are performed through a colonoscopy or upper endoscopy.
Ferring picked up the treatment in the 2018 acquisition of Minnesota-based Rebiotix. This September, an FDA advisory committee voted 13-4 to recommend Rebyota for an approval, which would help standardize treatment.
“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,” member Eric Rubin told the committee at the time.
The FDA thumbs-up was based on a review of data from six studies that spanned 1,061 participants, 978 of whom where treated with Rebyota. Of those on the therapy, 70% were free of infection within eight weeks as opposed to 58% in the placebo group, while safety profiles were comparable.
In gaining the approval, Switzerland-based Ferring beat out Seres Therapeutics, which applied for approval of its microbiome therapy and expects a decision in April of 2023.