C. difficile is emerging as a leading cause of life threatening, health-care acquired infections worldwide. © CDC

The decision of the vaccine arm of French drug giant Sanofi was based on an independent interim analysis of a pivotal Phase III study enroling up to 16,500 people at risk of Clostridium difficile infection (CDI) for vaccination with the jab that has FDA Fast Track designation.

Picture © OPIS

OPIS   Accelerated approval for orphan drugs and the possibility to have market authorisation
after a successful Phase II trial have made research in rare diseases more attractive to sponsors.
However, challenges and uncertainties remain numerous and designing scientifi cally robust,
patient-centered trials requires proper conceptualisation.

SHP647 mode of action: the antibody blocks integrin on B- and T-lymphocytes to attach to endothelial protein MAdCAM, which would translocate the leukocytes to the GI tract. © https://doi.org/10.2147/BTT.S71679

Shire has received FDA Orphan Drug Status for its anti-MAdCAM Antibody SHP647 to treat pediatric ulverative colitis.