The cord blood-based cell therapy quickens the recovery of neutrophils (a subset of white blood cells) and reduces the risk of infection.
As well as becoming a new donor source for allogeneic stem cell transplant, the treatment may increase access to stem cell transplants for patients for diverse backgrounds (who have a much lower chance of finding a match in a donor registry).
'Important advance in cell therapy treatment in patients with blood cancers'
Omisirge has been approved for use in adult and pediatric patients 12 years and older with hematologic malignancies planned for umbilical cord blood transplantation following myeloablative conditioning (such as radiation or chemotherapy) to reduce the time to neutrophil recovery and the incidence of infection.
“This approval is an important advance in cell therapy treatment in patients with blood cancers,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research.
Stem cell transplantation is a common treatment for blood cancers: putting healthy stem cells into the body to help restore the normal production and function of blood cells.
One source of healthy stem cells is umbilical cord blood.
Generally, before receiving this kind of transplant, the patient will undergo a course of treatments to remove their own stem cells and prepare the body for the new stem cells. This process may include undergoing therapies such as radiation or chemotherapy, both of which may weaken an individual’s immune system. As a result, a frequent and serious risk of this treatment is the occurrence of severe and sometimes deadly infections.
Omisirge, administered as a single intravenous dose, is composed of human allogeneic stem cells from umbilical cord blood that are processed and cultured with nicotinamide (a form of vitamin B3). Each dose is patient-specific, containing healthy stem cells from an allogeneic pre-screened donor, meaning it comes from a different individual rather than using the patient’s own cells.
“Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation.
"This approval reflects the FDA’s continued commitment to supporting development of innovative therapies for life-threatening cancers.”
Omisirge received breakthrough therapy designation, priority review and orphan drug designation from the FDA, reflecting the unmet need for additional donor sources for stem cell transplant.
It represents the first approval for the cell therapy company, which uses its proprietary nicotinamide (NAM) technology to leverage the properties of NAM to enhance and expand cells to create allogeneic cell therapy products and candidates for hematologic malignancies.
“FDA approval of Omisirge is a major advancement in the treatment of patients with hematologic malignancies that we believe may increase access to stem cell transplant and help improve patient outcomes,” said Abbey Jenkins, President and CEO of Gamida Cell.
“We are grateful to all the clinicians, patients and the entire Gamida Cell team without whom this approval would not have been possible.
"We also acknowledge the key role the FDA has played in supporting the development of Omisirge and other innovative and potentially life-saving cell therapies for patients with cancer and other serious diseases.”
Omisirge is the first allogeneic stem cell transplant therapy to be approved on the basis of a global, randomized Phase 3 clinical study.
In the study, Omisirge demonstrated a median time to neutrophil recovery of 12 days in the intent to treat population, compared to 22 days for standard cord blood (p<0.001). Incidence of Grade 2/3 bacterial or Grade 3 fungal infections through 100 days following transplantation occurred in 39% of patients in the Omisirge arm and 60% of patients in the standard cord blood arm. The full Phase 3 clinical study results are available in Blood, the official journal of the American Society of Hematology.
Increasing access for diverse patient populations
Steven M. Devine, M.D., Chief Medical Officer at the National Marrow Donor Program/Be The Match, describes Omisirge’s approval as a ‘significant development’ in hematopoietic stem cell transplantation.
“Patients who are Black or African American have just a 29% chance of finding a match via the donor registry vs. a 79% chance for patients who are White. Adding Omisirge as a new donor source may help increase access to stem cell transplant for patients from racially or ethnically diverse backgrounds who struggle to find a fully matched donor in the registry.”
More than 40% of the patients in the Omisirge Phase 3 study were racially and ethnically diverse.
Manufacturing base in Israel
Allogeneic hematopoietic stem cell transplantation offers a potentially curative option for hematologic malignancies including acute myeloid leukemia, acute lymphoblastic leukemia, chronic myeloid leukemia and myelodysplastic syndromes.
Omisirge is manufactured to enhance and expand the number of progenitor cells utilizing proprietary nicotinamide (NAM) technology. This process produces enriched hematopoietic progenitor cells, leading to preservation of their stemness, homing to the bone marrow and retained engraftment capacity.
Omisirge is manufactured in Gamida Cell’s fully licensed GMP manufacturing facility in Kiryat Gat, Israel: and can be delivered to transplant centers within 30 days after the start of manufacturing.
Gamida Cell Assist will act as a 'key resource' for scheduling the manufacturing of Omisirge and provide support to patients, caregivers and the hospital’s transplant team at each step of the process.
Omisirge is now available in the US for transplant centers to order for appropriate patients. Gamida Cell says it is pursuing strategic partnerships to support the launch and commercialization of Omisirge.