Devastating infectious disease gets a new FDA approved first-of-
its-kind treatment

“I had gone to a dentist for a tooth infection, and she put me on an antibiotic, which was necessary,” Pyles said. “That week, we were traveling to Kansas to visit relatives, and I developed diarrhea and was concerned about it. I kept taking the antibiotic, knowing that you’re supposed to take the whole course. The diarrhea kept getting worse and worse. It was just miserable.”3

A few hours into the drive home, Pyles collapsed outside a truck stop restroom. Her next stop was the nearest emergency room. She was critically dehydrated. Her kidneys were failing. Her blood pressure had plummeted. She had an infection called Clostridioides difficile, or C. diff—the most common healthcare associated infection in U.S. hospitals.1

C. diff infection isn’t just a stomach bug. Each year, the infection—declared an urgent public health threat by the U.S. Centers for Disease Control and Prevention—strikes about half a million Americans and takes 30,000 lives. Signaled by symptoms that can include stomach pain, nausea and diarrhea that may be of life-threatening severity, a C. diff infection can affect anyone. Older adults and people who are immunocompromised are more susceptible.4

According to Dr. Carl Crawford, a gastrointestinal specialist at Weill Cornell Medicine, the lower intestine is populated by a thriving community consisting of trillions of organisms. This diverse ecosystem—called the gut microbiome—plays a vital role in human health by producing vitamins B and K, facilitating nutrient absorption, enhancing immunity and more. Crucial to the microbiome’s functioning is the ecosystem’s balance.5-6

“Antibiotics are given to treat or prevent a specific infection,” Crawford said, but they sometimes can cause collateral damage in the gastrointestinal tract, leaving a susceptible individual at risk of dysbiosis—a shift in the microbiome where good bacteria die off and bad bacteria take over.7

Antibiotics can be a significant risk factor for C. diff infection, Crawford said. Within eight weeks of the initial diagnosis, up to one third of people who have recovered from C. diff infection may experience a recurrence.8,9,1

Pyles was among them. For four months, she underwent treatment with different antibiotics, but serious symptoms kept returning. She was cold and weak. And she couldn’t be more than a few feet from the bathroom. She certainly couldn’t care for her garden or her bees.

“I would have a break where I’d think I was over it,” she recalled. “I was so excited to be cured, or I thought I was cured. And then, wham, it would come back. I would think, ‘I can’t do it again.’ I told my husband, ‘If I get it again, I’m not going to live.’ I really thought if I got C. diff one more time, I wasn’t going to make it.”

Crawford and his team at Weill Cornell are very familiar with the way the physical symptoms of C. diff infection can upend lives.

“It’s very sad when I have patients who are ostracized by their family because this is an infectious disease and it can be transmitted,” Crawford said. “We have a lot of grandparents who aren’t allowed to see their grandchildren. You have parents whose children don’t want to see them. You have individuals whose friends don’t want to come over to see them. It can be a very isolating disease.”

Many of the patients Dr. Crawford sees in his practice have residual symptoms, even after treatment. “They never quite feel the same afterwards,” Crawford said. In his experience, he has seen some patients come in with new food intolerances and symptoms resembling the potential first signs of a recurring C. diff infection that can lead to weight loss, depression and a syndrome Crawford likens to post-traumatic stress disorder.10

To better address C. diff infection recurrences and residual symptoms, some pharmaceutical companies are experimenting with antibiotics that have a narrower spectrum of activity — agents designed to potentially only attack C. diff. Other companies, like Ferring Pharmaceuticals, are taking a different approach, looking at the link between the gut microbiome and recurrent C. diff.

Four months into her experience with recurrent C. diff infection, Pyles learned about Ferring’s investigational therapy. She sought treatment at Yale - New Haven Hospital. After receiving standard-of-care antibiotic treatment, she received an investigational microbiota-based treatment.

Recently, the Food and Drug Administration (FDA) approved the microbiota-based treatment that Pyles received at the hospital. The treatment, Ferring’s REBYOTA™ (fecal microbiota, live—jslm), is a first-in-class, single-dose, microbiota-based live biotherapeutic indicated for the prevention of recurrence of Clostridioides difficile (C. diff) infection in individuals 18 years of age and older, following antibiotic treatment for recurrent C. diff infection. In clinical trials, REBYOTA was well-tolerated with generally expected and manageable adverse reactions. Adverse reactions were mostly mild to moderate in severity and the most common adverse reactions were diarrhea, abdominal pain and nausea.

Over the past decade, Ferring has been working to develop novel microbiome-based biotherapeutics to help address significant unmet needs, starting with recurrent C. diff infection.