Alexion Pharmaceuticals Inc.'s phase 2/3 trial of eculizumab did not meet its primary endpoint.
The trial evaluated eculizumab for the prevention of delayed graft function after kidney transplantation in adult recipients of a deceased donor kidney. Delayed graft function is a complication in organ transplants where the transplanted organ fails to function normally immediately after the transplant, and was defined in the study as the requirement for dialysis for any reason in the first seven days after the transplant.
The primary endpoint of incidence of delayed graft function with a two-dose regimen of eculizumab, compared with placebo, did not reach statistical significance. In the primary endpoint of the study, assessed in 286 patients who were treated and received a transplant, the incidence of delayed graft function, death, graft loss, or loss to follow-up at seven days post-transplant was 35.9% for patients receiving a two-dose regimen of eculizumab compared with 41.7% for patients receiving placebo.
For the first 60 days after treatment, rates of serious adverse events were higher for patients in the placebo group compared with the eculizumab group — 54.1% compared to 44.4%. There were four deaths in the placebo group compared with one death in the eculizumab group in the same time frame.
Eculizumab previously received an orphan drug designation from the U.S. FDA in 2014 for the prevention of delayed graft function in renal transplant patients, and from the European Medicines Agency for the prevention of delayed graft function after solid organ transplantation. The drug is not approved in any country for the treatment or prevention of delayed graft function.