RARE Daily

FDA Grants Eligo Rare Pediatric Disease Designation for Crisper-based Therapy for Hemolytic Uremic Syndrome

October 11, 2022

The U.S. Food and Drug Administration granted Eligo Bioscience Orphan Drug and Rare Pediatric Disease designations for its oral drug candidate EB003, for the treatment of Shiga-toxin producing bacterial infection as it relates to the prevention of hemolytic uremic syndrome.

Photo: Xavier Duportet, CEO of Eligo Bioscience

Children under 5 years of age are particularly sensitive to the expression of Shiga toxins from E. coli (STEC) bacteria after their ingestion from contaminated foods. Toxin production in the gut triggers bloody diarrhea, and its translocation and accumulation in the systemic compartment can lead to hemolytic uremic syndrome (HUS), a life-threatening condition involving destruction of blood platelets, anemia, and acute kidney injury. There are currently no approved therapies for this rare disease.

EB003 is a first-in-class microbiome gene therapy designed, built, and optimized to target STEC bacteria in the gut of infected patients, leveraging Eligo’s unique expertise in synthetic biology, phage biology, genetic engineering, and bioinformatics. As opposed to antibiotics which unfortunately lead to Shiga toxin overproduction in the process of killing the STEC bacteria, Eligo’s proprietary sequence-specific CRISPR technology mechanism of action leads to the disruption of the genes coding for the Shiga toxin.

“Granting of Orphan Drug designation and Rare Pediatric Disease designation for EB003 highlights the FDA’s recognition of the potential of how our unique CRISPR-based modalities can be used to address devastating diseases driven by the expression of bacterial genes, such as hemolytic uremic syndrome,” said Xavier Duportet, CEO of Eligo Bioscience.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases primarily affecting individuals ages 18 or younger and fewer than 200,000 individuals in the United States. If EB003 is approved by the FDA, Eligo Bioscience may be eligible to receive a priority review voucher, which can be used to reduce the time for a subsequent application to a six-month review from a 10-month review. redemption of which will result in priority review for any subsequent marketing application. The PRVs are potentially lucrative because they are transferrable. In July, Marinus sold a priority review voucher to Novo Nordisk for $110 million.

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