Omidubicel is an advanced NAM-enabled stem cell therapy candidate which is being developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for blood cancer patients.
It is claimed to be the first bone marrow transplant product to receive Breakthrough Therapy Designation from the US Food and Drug Administration.
The product has also received Orphan Drug Designation in both the European Union (EU) and the US.
The company was granted priority review, with a Prescription Drug User Fee Act (PDUFA) action date of 30 January next year.
The BLA submitted to the FDA was supported by significant data obtained from the company’s pivotal Phase III study, which was conducted in hematologic malignancies patients who are undergoing allogeneic bone marrow transplant.
In the study, omidubicel demonstrated a statistically significant reduction in median time to neutrophil engraftment compared with standard umbilical cord blood.
It also demonstrated reduced time to platelet engraftment, reduced infections, as well as the days of hospitalisation.
Gamida Cell CEO Julian Adams said: “The FDA’s acceptance of our BLA with Priority Review signifies a critical milestone in our mission to deliver a new stem cell therapy option for patients in need of a donor for an allogeneic stem cell transplant.
“We are encouraged by the positive and sustained follow-up results from patients participating in the Phase 3 trial of omidubicel, including a positive overall survival trend one-year out from treatment.
“These results provide promising rationale that, if approved, omidubicel could become a treatment of choice for patients in need of an allo-HSCT transplant.”
The company plans to manufacture omidubicel at its manufacturing facility in Israel after receipt of FDA approval.
The newly constructed, advanced modular facility also has the capacity to handle growth and demand for the product.