November 04, 2024
2 min read
As of Nov. 1, OpenBiome suspended its distribution of investigational fecal microbiota transplantation, which may put certain patients with recurrent, refractory or fulminant Clostridioides difficile infection at risk, according to the AGA.
“It is important to understand that the suspension is a voluntary action on the part of OpenBiome, which is the result of some communications they have received from the FDA,” AGA spokesperson Colleen R. Kelly, MD co-principal investigator of the AGA’s FMT National Registry and gastroenterologist at Brigham and Women’s Hospital, told Healio. “FDA issued a guidance in November 2022, which stated that enforcement policy around investigational new drug applications for FMT would no longer be extended to ‘stool banks.’”
!["[If the] FDA does not continue to allow enforcement discretion for stool banks, many of these patients will lose access to FMT, and some may die.” Colleen R. Kelly, MD](https://www.healio.com/~/media/slack-news/gastroenterology/misc/infographics/2024/1124/hgi1124aga_fmtletter_graphic_01.jpg?w=800)
According to an OpenBiome press release, the FDA allowed the nonprofit organization to continue shipment of investigational FMT produced by the University of Minnesota through Oct. 31, 2024, during which time OpenBiome would accelerate its timeline for an IND submission.
Kelly noted that OpenBiome has spent “over a year” trying to find a solution for this updated policy, including through the FDA’s Real World Evidence Program.
With no solution in sight, the AGA, in collaboration with the Infectious Diseases Society of America, NASPGHAN, the Crohn’s and Colitis Foundation and the Peggy Lillis Foundation, sent a letter to Peter Marks, MD, PhD, head of the FDA’s Center for Biologics Evaluation and Research, urging the agency to “retain an enforcement policy that would preserve access to FMT” for patients for whom currently approved therapies are not indicated or are ineffective or difficult to access.
In addition, the organizations requested the opportunity to meet with FDA representatives to discuss current evidence, recommendations and clinical practice trends related to FMT.
“We do not currently have an FDA-approved FMT product for severe or fulminant C. difficile or for pediatrics,” Kelly said. “Furthermore, there are already patients who have failed to resolve their infections with the available products. [If the] FDA does not continue to allow enforcement discretion for stool banks, many of these patients will lose access to FMT and some may die.”
Although the letter highlighted the FDA’s recent approval of Rebyota (fecal microbiota, live-jslm; Ferring Pharmaceuticals) and Vowst (fecal microbiota spores, live-brpk; Nestlé Health Science/Seres Therapeutics) for prevention of recurrent C. difficile infection in adult patients who have completed antibiotic therapy, the authors noted that recurrent, refractory and fulminant infections “continue to pose a challenge” for clinicians and patients. For this reason, conventional FMT from known donors or stool banks remains an “essential, safe and effective” therapeutic option, they wrote.
“The currently approved FMT therapeutics largely excluded immunocompromised patients from their clinical trials, so it is unknown how effective or safe these will be in this population,” Kelly told Healio. “AGA published FMT guidelines earlier this year that recommended ‘conventional FMT’ rather than either Rebyota or Vowst for these patients. These formulations are also much more expensive, and depending on the type of insurance a patient has, may cost thousands of dollars per dose or treatment course.”
According to Kelly, the AGA also will continue to support the FMT National Patient Registry to generate real-world data on the procedure’s safety and efficacy and will intensify efforts to enroll patients with severe and fulminant infections.
References: