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Gut Instinct: Rebyota

Pharmaceutical ExecutivePharmaceutical Executive: September 2023
Volume 44
Issue 9

Bacteria killer signals a milestone—and momentum—for microbiome therapeutics.


If there is one thing we can all agree is awful, it’s bacteria and viruses that cause projectile anything, if you pick up what I’m putting down. Anyone who has had Clostridioides difficile (C. diff) knows how debilitating it is, and anyone who has worked in healthcare can attest to the contagious nature of this bacteria. Because C. diff is a gift that keeps on giving, recurrent C. diff can cause severe diarrhea, fever, stomach tenderness/pain, loss of appetite, nausea, and colitis. On behalf of all humans, there is now something out there to treat recurrence. Because for the sake of all that is good, having it one time is more than sufficient.

Rebyota (fecal microbiota, live-jslm), developed by Ferring Pharmaceuticals, is the first and only FDA-approved microbiota-based live biotherapeutic that works to prevent the recurrence of C. diff infection (CDI) at first recurrence. Launched in January 2023, the drug is for adults 18 years and older following antibiotic treatment for recurrent CDI. It is not indicated for the treatment of CDI.

Lionel Fajolle

Lionel Fajolle

Administered rectally by a healthcare professional, this microbiota suspension delivers live microbes—including Bacteroides—in one 150-mL dose. While the idea of rectal administration may be unappealing, this treatment doesn’t require bowel prep, anesthesia, or a colonoscopy, which is a win. Moreover, for ease of traceability, each stool sample has a single donation from a healthy donor. In other words, donations are not pooled.

“The efficacy and safety of Rebyota were studied in the largest clinical trial program ever conducted in the field of gut microbiome-based therapeutics, including five clinical trials with more than 1,000 participants,” says Lionel Fajolle, vice president, specialty care, Ferring Pharmaceuticals US, tells Pharmaceutical Executive®. “Results from the randomized, double-blind Phase III PUNCH CD3 trial showed a treatment success rate of 70.6% at eight weeks for Rebyota vs. 57.5% for placebo. More than 90% of participants who achieved treatment success remained free of CDI recurrence for six months. The most common side effects of Rebyota may include stomach pain (8.9%), diarrhea (7.2%), bloating (3.9%), gas (3.3%), and nausea (3.3%).”

Historically, the standard treatment for CDI is antibiotics. However, this treatment can contribute to recurrence since—while it kills or stops the growth of C. diff bacteria—it can also kill good bacteria. Thus, antibiotics can lead to disruption in the microbiome and CDI recurrence.

“Rebyota represents a major breakthrough in harnessing the power of the gut microbiome to address a significant unmet medical need. Rebyota takes a revolutionary approach to preventing recurrent [CDI], with each single, pre-packaged dose delivering potentially trillions of live microbes directly where it is needed, the gut microbiome,” says Fajolle.

But like all new therapeutics, in order to make an impact, providers and patients must first know that this medicine is available to them and then be able to afford it. Ferring Pharmaceuticals, according to Fajolle, offers a patient support platform, which includes copay and patient assistance programs. The platform also has disease and product information.

“Well before our FDA approval, we identified and made a deliberate choice to address a significant microbiome knowledge gap that existed with patients as well as practitioners,” says Fajolle. “I am proud to say that our disease state educational content has been equally praised by many healthcare providers and C. diff patient associations.”

But what’s next for Rebyota? According to Fajolle, the company is looking to see where microbiomes may have the potential to treat disease areas beyond recurrent C. diff.

“We remain focused on changing the standard of care for patients suffering from the burden of recurrent C. diff infection and ensuring those who need Rebyota have access to it. We continue to look at broader, real-world patient populations that are seen in clinical practice and look forward to presenting or publishing data when appropriate,” says Fajolle. “In addition, we are looking at disease areas beyond recurrent C. diff; and as a pioneer in the microbiome space, we continue to explore the crucial link between the gut microbiome and human health.”

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