BOSTON–(BUSINESS WIRE)–Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, today announced that it has initiated the Biologics License Application (BLA) rolling submission process with the U.S. Food and Drug Administration for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant. The company remains on track to complete the BLA submission in the second quarter of 2022.
“We are pleased to reach this important milestone for omidubicel and bring this potential therapy one step closer to reaching patients in need,” said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. “In the Phase 3 study, omidubicel achieved a statistically significant reduction in time to neutrophil engraftment, reduced hospitalization time, decreased risk of infection and shorter time to platelet engraftment. Based on this positive data, we believe omidubicel has the potential to address the existing unmet needs in allogeneic transplant, offering a new standard of care and the opportunity to treat even more patients.”
Omidubicel has the potential to be the first FDA approved advanced cell therapy product for allogeneic stem cell transplant. For patients with hematologic malignancies that are deemed eligible for an allogeneic stem cell transplant, the procedure is their best chance for a potential cure. In the U.S., there are approximately 8,000 patients above the age of 12 with hematologic malignancies who undergo an allogeneic stem cell transplant each year and we believe that number of patients may grow over time1. Unfortunately, there are approximately 1,000 patients each year, who are above the age of 12 and are deemed eligible for an allogeneic stem cell transplant but cannot find an appropriate donor2. Based on its encouraging clinical data and less stringent matching criteria, omidubicel has the potential to improve outcomes for allogeneic stem cell transplant patients compared to other donor sources and expand access for patients who cannot find a suitable donor.
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with blood cancers. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the U.S. FDA and has also received Orphan Drug Designation in the U.S. and EU. Gamida Cell has completed an international, multi-center, randomized Phase 3 study (NCT0273029) evaluating the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing allogeneic bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. That study achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in a patient’s recovery from a stem cell transplant. The Phase 3 study also achieved its secondary endpoints of reduced time to platelet engraftment, reduced infections and shorter days of hospitalization. For more information about omidubicel, please visit https://www.gamida-cell.com.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About Gamida Cell
Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.