Gamida Cell Ltd. announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s Biologics License Application (BLA) for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations.
The FDA granted Priority Review for the BLA and has set a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2023. The FDA grants Priority Review to product applications that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. At this time, the FDA has indicated that it is not planning an advisory committee meeting as part of the BLA review.
“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,” said Julian Adams, Ph.D., chief executive officer of Gamida Cell. “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. We look forward to working with the FDA throughout the review process to bring omidubicel to patients as quickly as possible.”
Upon FDA approval, omidubicel will be manufactured at the Gamida Cell owned manufacturing facility in Israel. This is a newly constructed, state-of-the-art, modular facility which allows for additional capacity to be added to address growing demand. Batches from this facility were used to support the BLA for omidubicel and the facility is currently manufacturing clinical batches.
The omidubicel BLA is supported by the statistically significant results from Gamida Cell’s pivotal Phase 3 study, the results of which were published in Blood, the official journal of the American Society of Hematology. Results for the study’s primary endpoint, the median time to neutrophil engraftment in patients with hematologic malignancies undergoing allogeneic bone marrow transplant with omidubicel compared to standard umbilical cord blood (UCB), demonstrated a median time to neutrophil engraftment of 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p < 0.001). The secondary endpoints of this Phase 3 study were all achieved and were statistically significant. These secondary endpoints were platelet engraftment, the rate of infection, and days alive and out of hospital. Omidubicel was generally well tolerated in the Phase 3 study.