If approved the drug will be the first allogeneic advanced stem cell therapy donor source for patients with blood cancers.
The FDA has accepted filing for Gamida Cell Ltd’s biologics license application (BLA) for omidubicel for the treatment of individuals with blood cancer who need an allogenic hematopoietic stem cell transplant (allo-HSCT).
Omidubicel is an advanced, first-in-class NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designation from the FDA. It is also the first stem cell transplant donor source to received breakthrough therapy designation from the FDA, as well as orphan drug designation in the European Union and the United States.
The FDA granted priority review for the BLA and has set a Prescription Drug User Fee Act target date of January 30, 2023.
“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,” Julian Adams, PhD, CEO of Gamida Cell, said in a statement.
“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 1-year out from treatment,” Adams said. “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 FDA grants priority review to product applications that, if approved, would provide significant improvements in diagnosis, effectiveness or safety of treatment, or prevention of a serious condition. The FDA has indicated that it is not planning an advisory meeting as part of the BLA review.
The BLA is supported by the results from a pivotal phase 3 study published in Blood. The findings showed that the study’s primary endpoint, the median time to neutrophil engraftment in individuals with hematologic malignancies undergoing allogeneic bone marrow transplant with omidubicel compared with standard umbilical cord blood, demonstrated a median time of 12 days for all individuals who were randomized to omidubicel compared with 22 days for those taking the comparator.
Additionally, the second endpoints of the phase 3 study, including days alive and out of hospital, platelet engraftment, and rate of infection, were all achieved and were statistically significant.
Furthermore, 1-year post-transplant data showed sustained clinical benefits with the drug demonstrated by a significant reduction in infectious complications, as well as reduced non-relapse mortality, and there was no significant increase in relapse rates or in graft-versus-host-disease rates.
Omidubicel was generally well tolerated in the study.
If approved, omidubicel will be manufactured at Gamida Cell’s manufacturing facility in Israel. Batches from the facility were used to support the BLA for the drug and the facility is manufacturing clinical batches.
If approved, omidubicel has the potential to treat approximately 2000 to 2500 individuals each year in the United States alone and could improve outcomes based on transplanter feedback and increase access to transplants, according to the statement.
Gamida cell announces FDA acceptance of biologics license application for omidubicel with priority review. News release. Gamida Cell. August 1, 2022. Accessed August 2, 2022. https://investors.gamida-cell.com/news-events/press-releases/news-release-details/gamida-cell-announces-fda-acceptance-biologics