Last week, the US Food and Drug Administration (FDA) quietly announced it would be backing off a previous proposal to require fecal microbiota transplants to be conducted under either an approved biologics license application (BLA) or investigational new drug (IND) application. Now, however, it has announced the approval of fast track designation for an investigational product sponsored by Minnesota-based Rebiotix, potentially one of the first - if not the first - products to seek formal approval as an FMT product.
An FMT is a transplant of carefully-selected bacteria found in ones' colon to another person to fight Clostridium Diffiicile (C. Diff), a notoriously hardy strain of bacteria that kills an estimated 14,000 people each year.
Various published research has identified the FMT procedure as being one that can fight off C. diff infections by introducing so-called "good bacteria" back into a person's body. The idea is to overload the gastrointestinal tract with enough of the good bacteria to overwhelm the C. diff, thereby ending the infection.
Despite its promise, the area has been rife with regulatory difficulties. In recent months, FDA had sought to crack down on a mostly unregulated sector by announcing it would require INDs for the treatment of patients with FMT. Regulators said they were concerned that some doctors may introduce undesirable bacteria during the process, leading to further difficulties.
Doctors, however, fought back against the IND requirements, explaining that the added burdens of adhering to clinical trials regulations would make the therapy more difficult, more expensive, more time-consuming, and ultimately drive more patients to engage in do-it-yourself fecal transplants.
In June 2013, FDA quietly announced it would do away with the requirement, seeking to instead adopt "enforcement discretion" over the sector. In other words, it generally will not regulate FMT procedures, but retains the right to enforce the sector if it believes a doctor is subjecting his or her patients to harmful treatments.
Fecal Fast Track
Until now, however, FDA has been dealing with FMT as a procedure, and not a distinct product. Now, though, the agency has granted "fast track" status to Rebiotix' RBX2660, a microbiota suspension intended to treat C. Diff infections.
Fast track designation is given to a drug application for which there is evidence of the potential to address an unmet medical need or one that has been designated as a qualified infectious disease product, the latter being a new condition established under the Generating Antibiotic Incentives Now (GAIN) Act. The designation allows FDA to receive application materials on a rolling basis, and for regulators to expedite the development and review of a product.
In a statement announcing the status, Rebiotix said the designation "reinforces the urgent need for a new therapy for recurrent CDI to treat patients who have this debilitating and potentially life-threatening disease. Patients with recurrent CDI often have to endure months of illness and only feel better when they are taking antibiotics. Once the antibiotic therapy stops, the disease process often begins again, disrupting their lives."
It is unclear as to what effect an eventual approval (if it occurs) of Rebiotix' application would have on FMT procedures. Typically an approved application carries with it several years of market exclusivity, but if the company meets GAIN Act conditions, it would be available for additional exclusivity as well. This would prevent competitors from marketing the same combination of FMT for the same C. Diff claim, but would likely not stop doctors from in effect "compounding" their own FMT products for use by their patients.
As FMT is a combination of a large number of bacteria, it also remains possible that another company could obtain approval for a variant of the combination.
Rebiotix Press Statement