FDA’s approval of first fecal microbiota product, Rebyota, hailed an ‘enormous step forward’ for the field

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The first fecal microbiota product, Rebyota, approved by the U.S. Food and Drug Administration (FDA) today has been hailed ‘fantastic news’ by those in the microbiome field.

Ferring Pharmaceutical’s product is approved to stop Clostridiodes difficile infection (CDI) in people over 18 and to be given after they have completed an antibiotic treatment for the infection.

Brent Ragans, president, Ferring Pharmaceuticals U.S said: “Rebyota is a much-needed new treatment that offers hope to the thousands of people who suffer from recurrent C. difficile infection each year. It has the potential to impact not only patients and caregivers, but also the healthcare system.”

James McIroy is CEO of EnteroBiotix,  a company he founded with the vision of building one of the world’s leading microbiome therapeutics businesses. He believes ‘everyone on the planet’ would benefit from enhancing their microbiome.

He said: “The FDA approval of Rebyota is fantastic news for the microbiome therapeutics field. It shows recognition by regulators of how it can really make a difference in hard-to-treat, and potentially life-threatening diseases, such as C. diff infection, and could transform the standard of care. It strongly reinforces the centuries old philosophy that gut health is health.

“More broadly, I think that every person on the planet can benefit from enhancement of their microbiome. For patients that are unwell and that have microbiome damage, I think approaches like Rebyota will be highest yield, for others, I believe that diet and lifestyle changes will be enough to be highly impactful.”

Rebyota – the highest yield

Clostridioides difficile (C. difficile) is a bacterium that can cause CDI, a potentially life-threatening disease resulting in diarrhea and significant inflammation of the colon. In the United States, CDI is associated with 15,000-30,000 deaths annually.  

The intestinal tract contains millions of microorganisms, often referred to as the “gut flora,” or “gut microbiome.” Certain situations, such as taking antibiotics to treat an infection, may change the balance of microorganisms in the gut, allowing C. difficile to multiply and release toxins causing diarrhea, abdominal pain and fever, and in some cases, organ failure and death.

Hervé Affagard, CEO of Maat Pharma, the company that Labiotech wrote about in November, said: “Today marks a major milestone in the global microbiome industry as we see the first FDA-approved microbiome product. The first but certainly not the last! With this significant regulatory milestone for microbiome therapies, we expect a significant acceleration of the field.

“This is also an enormous step forward for the patients as this addresses a high unmet medical need. As the president of Alliance Promotion Microbiote, an association including more than 20 key French players of the microbiome industry, I would also like to add that France has been defining a comprehensive regulatory framework since 2015 and that we firmly believe that members of Alliance Promotion Microbiote have the potential to have products approved soon.

Restoration of gut flora

Other factors that can increase the risk for CDI include age older than 65 years, hospitalization, a weakened immune system and a previous history of CDI. After recovering from CDI, individuals may get the infection again—often multiple times—a condition known as recurrent CDI. The risk of additional recurrences increases with each infection and treatment options for recurrent CDI are limited. The administration of fecal microbiota with a product such as Rebyota is thought to facilitate restoration of the gut flora to prevent further episodes of CDI.

“Today’s approval of Rebyota is an advance in caring for patients who have recurrent C. difficile infection,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research.

“Recurrent CDI impacts an individual’s quality of life and can also potentially be life-threatening. As the first FDA-approved fecal microbiota product, Rebyota, announced in yesterday’s action represents an important milestone, as it provides an additional approved option to prevent recurrent CDI.”

Rebyota manufactured from human feces

The fecal microbiota product is administered rectally as a single dose. It’s prepared from stool donated by qualified individuals. The donors and the donated stool are tested for a panel of transmissible pathogens.

Bjørn Nielsen, CSO at Clinical Microbiomics, added: “It is incredibly exciting that Rebyota has received approval by the FDA. It is indeed an achievement for Rebiotix and Ferring, but also for the whole field of microbiome-based therapeutics, and even microbiome as such.

“Recurrent CDI is a deadly infectious disease, and I think it has been clear to many in the microbiome field that treatment like this could be a solution. I am convinced this first microbiome-based treatment will help many patients. It is very significant, and I am sure this will encourage the entire microbiome field going forward. Clinical Microbiomics is committed to supporting companies in the microbiome space to achieve similar goals.”

However, there are a couple of potential risks as Rebyota is manufactured from human fecal matter. It may carry a risk of transmitting infectious agents. Also it could be risky for people with food allergies as Rebyota may contain food allergens. The potential for the product to cause adverse reactions due to food allergens is unknown.

Common side-effects diarrhea, gas and bloating

The safety of Rebyota was assessed from two randomized, double-blind, placebo-controlled clinical studies and from open-label clinical studies conducted in the United States and in Canada. The participants had a history of one or more recurrences of CDI. They received one or more doses of Rebyota or placebo 24 to 72 hours after completion of antibiotic treatment for their CDI. Participants’ CDI was under control at the time of receipt of Rebyota or placebo.

Across these studies, 978 individuals aged 18 years and older received at least one dose of Rebyota. In one study, among 180 Rebyota recipients, when compared to 87 placebo recipients, the most common side effects after receiving one dose of Rebyota were abdominal pain, diarrhea, abdominal bloating, gas and nausea.

The effectiveness of Rebyota was evaluated in an analysis of data from a randomized, double-blind, placebo-controlled, multicenter study. The analysis included 177 adults who received one dose of Rebyota and 85 who received one dose of placebo in this study. It also incorporated success rates from a different placebo-controlled study in which 39 adults received one dose of Rebyota and one dose of placebo and 43 adults received two doses of placebo. Success in preventing recurrent CDI was defined as the absence of CDI diarrhea within 8 weeks of administration of Rebyota or placebo. In a statistical analysis that took into account both studies, the overall estimated rate of success in preventing recurrent CDI through 8 weeks was significantly higher in the Rebyota group (70.6%) than in the placebo group (57.5%).

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