BOSTON–(BUSINESS WIRE)–Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-compatible cell therapy candidates for patients with hematological and solid cancers and other serious diseases, today announced the completion of the license application submission (BLA) to the United States Food and Drug Administration (FDA) for omidubicel for the treatment of patients with blood cancers requiring allogeneic hematopoietic stem cell transplantation.
“The BLA submission marks an important milestone for Gamida and the transplant community, as omidubicel has the potential to be the first advanced cell therapy product approved for allogeneic stem cell transplantation,” said Julian Adams, Ph. D., General Manager of Gamida Cell. “The completion of this BLA submission is a key inflection point in our mission to provide a new treatment option for patients with blood cancers. We look forward to working closely with the FDA to bring this potentially important therapy to patients. »
The FDA has 60 days to determine if the BLA for omidubicel is acceptable for filing. Omidubicel BLA builds on statistically significant results from Gamida Cell’s pivotal Phase 3 study, the results of which have been published in Blood, the official journal of the American Society of Hematology. For the study’s primary endpoint, the median time to neutrophil engraftment in patients with hematological malignancies undergoing allogeneic bone marrow transplantation receiving omidubicel versus cord blood umbilical cord (UCB), the median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p
In the primary secondary endpoints of this phase 3 study: Platelet engraftment was significantly accelerated [55 percent of patients randomized to omidubicel achieving platelet engraftment by day 42, compared to 35 percent for the comparator (p = 0.028)]; the infection rate was significantly reduced [cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.03)]; and hospitalization in the first 100 days after transplant was significantly reduced [median number of days alive and out of hospital for patients randomized to omidubicel of 61 days, compared to 48 days for the comparator (p = 0.005)]. Omidubicel was generally well tolerated in the phase 3 study.
Full Blood the manuscript is available here: https://ashpublications.org/blood/article/doi/10.1182/blood.2021011719/476235/Omidubicel-Versus-Standard-Myeloablative-Umbilical.
Omidubicel is an advanced cell therapy candidate developed as a potentially life-saving allogeneic hematopoietic (bone marrow) stem cell transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in neutrophil transplant time compared to standard umbilical cord blood in an international, multicenter, randomized Phase 3 study (NCT0273029) in patients with hematological malignancies undergoing allogeneic cord transplant. bone marrow. The phase 3 study also showed a reduction in platelet transplant time, a reduction in infections and fewer days of hospitalization. One-year post-transplant data showed durable clinical benefits with omidubicel, as evidenced by a significant reduction in infectious complications as well as a reduction in relapse-free mortality and no significant increase in relapse rates or increase in rate of graft versus host disease (GvHD). Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.
Omidubicel is an investigational therapy, and its safety and effectiveness have not been established by the FDA or any other health authority. For more information on omidubicel, please visit https://www.gamida-cell.com.
In 2019, approximately 8,000 patients aged 12 and over with hematological malignancies underwent allogeneic stem cell transplantation.1 Unfortunately, an estimated 1,200 other patients were eligible for a transplant but could not find a donor.2 Omidubicel, if approved, has the potential to improve patient outcomes based on transplantor feedback and potentially increase patient access to transplantation. Omidubicel, if approved, has the potential to treat approximately 2,000 to 2,500 patients each year in the United States
About NAM Technology
Our NAM technology is designed to improve the number and functionality of targeted cells, allowing us to pursue a healing approach that goes beyond what is possible with existing therapies. By taking advantage of the unique properties of NAM (nicotinamide), we can grow and metabolically modulate multiple cell types – including stem cells and natural killer cells – with appropriate growth factors to maintain the active phenotype of cells and improve their Powerful. Additionally, our NAM technology improves the metabolic fitness of cells, allowing continued activity throughout the expansion process.
About the Gamida Cell
Gamida Cell is at the forefront of a diverse immunotherapy portfolio of potentially curative cell therapy candidates for patients with solid tumors, blood cancers and other serious blood diseases. We are applying a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources, including umbilical cord blood-derived cells and NK cells, to create therapeutic candidates that have the potential to redefine the standard of care. These include omidubicel, an investigational product with the potential to save the lives of patients in need of bone marrow transplants, and a modified and unmodified NAM-activated NK cell line targeted to solid tumors and hematological malignancies. For more information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.
Caution Regarding Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to the timing of the initiation and progress of clinical trials of Gamida Cell’s product candidates (including including omidubicel), anticipated regulatory filings (including the timing of the BLA submission for omidubicel to the FDA), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of the product candidates of Gamida Cell (including omidubicel) and the expectations regarding anticipated clinical development milestones set forth herein. Any statement describing Gamida Cell’s objectives, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. These statements are subject to a number of risks, uncertainties and assumptions, including those relating to the impact the COVID-19 pandemic may have on our business, and including the scope, progress and expansion of Gamida Cell’s clinical trials and their ramifications for cost. whose ; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for human therapeutic use, and the effort of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties described in the Risk Factors section and other sections of Gamida Cell’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission (SEC ) on March 24, 2022, as amended, and other documents filed by Gamida Cell with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in these forward-looking statements may not occur, and Gamida Cell’s actual results could differ materially and adversely from those anticipated or implied. Although Gamida Cell’s forward-looking statements reflect the good faith judgment of its management, such statements are based solely on facts and factors currently known to Gamida Cell. Accordingly, you are cautioned not to rely on these forward-looking statements.
1CIBMTR 2019 – allogeneic transplants in patients aged 12 and over with hematological malignancies.
2Gamida Cell Market Research