Synthetic Biologics’ SYN-004 secures FDA breakthrough therapy status

Synthetic Biologics has secured breakthrough therapy designation for SYN-004 (ribaxamase) from the US Food and Drug Administration (FDA) to treat Clostridium difficile infection (CDI).

CDI is a hospital acquired infection that affects approximately 453,000 patients in the US every year, resulting in nearly 29,000 deaths.

SYN-004 is a first-in-class oral enzyme designed to degrade specific IV beta-lactam antibiotics within the GI tract to maintain the natural balance of the gut microbiome and prevent CDI.

Synthetic Biologics president and CEO Jeffrey Riley said: "We are delighted by the FDA's recognition of ribaxamase's potential to prevent CDI, and the dire need to fill the current void of an approved intervention.

"Following this announcement, we have been asked and anticipate requesting a Type-B multidisciplinary meeting with the Agency for a comprehensive discussion on the overarching, high-level drug development plan and pathway to licensure for ribaxamase.

"We look forward to working closely with the FDA throughout the development and review process and remain dedicated to bringing this potentially paradigm-shifting approach to antibiotic therapy to patients in critical need."

The FDA breakthrough therapy designation was awarded based on data from the successful Phase IIb clinical trial of ribaxamase.

Results from the trial showed that patients receiving ribaxamase achieved a 71.4% relative risk reduction in CDI rates.

It also demonstrated a significant reduction in new colonisation by vancomycin-resistant enterococci (VRE) for patients receiving ribaxamase compared to placebo.

If it receives the final approval from FDA, SYN-004 will be the first available drug designed to prevent Clostridium difficile infection by protecting the gut microbiome from antibiotic-mediated dysbiosis.