FDA approves Omisirge for allogeneic stem cell transplant therapy

Omisirge received breakthrough therapy designation, priority review and orphan drug designation from the FDA, reflecting the unmet need for additional donor sources for stem cell transplant. It is the first allogeneic stem cell transplant therapy to be approved on the basis of a global, randomized Phase III clinical study.

In a global, randomized Phase III clinical study, Omisirge demonstrated a median time to neutrophil recovery of 12 days in the intent to treat population, compared to 22 days for standard cord blood (p<0.001). incidence of grade 2 3 bacterial or grade 3 fungal infections through 100 days following transplantation occurred in 39% of patients in the omisirge arm and 60% of patients in the standard cord blood arm. the full phase iii clinical study results are available in blood, the official journal of the american society of hematology. the safety profile for omisirge is consistent with the expected adverse events of allogeneic hematopoietic stem cell transplantation following myeloablative conditioning. among 117 patients who received omisirge for any disease, infusion reactions occurred in 47% of patients (grade 3 or 4 in 15%), acute graft-versus-host disease (gvhd) in 58% (grade iii-iv in 17%), chronic gvhd in 35% and graft failure in 3%.

See-"Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase III randomized study": Mitchell E. Horwitz, Patrick J. Stiff, Corey Cutler, Claudio Brunstein, Rabi Hanna, Richard T. Maziarz, Andrew R. Rezvani, et al.,: Blood (2021) 138 (16): 1429–1440. https://doi.org/10.1182/blood.2021011719.