Priority Review of Gamida’s Novel Transplant Therapy Reflects Urgency, Efficacy

Ronit Simantov_Gamida Cell

Gamida CMO and CSO Dr. Ronit Simantov, M.D./Courtesy of Gamida Cell Ltd.

Boston-based Gamida Cell Ltd. announced Monday that the U.S. Food and Drug Administration has accepted its Biologics License Application for omidubicel. The novel therapy is being developed for the treatment of patients with blood cancers who need an allogeneic hematopoietic stem cell transplant.

Possibly reflecting the urgent need for an alternative donor source, the FDA granted Priority Review for the BLA with a target action date of January 30, 2023. Omidubicel, a first-in-class, advanced NAM (nicotinamide)-enabled stem cell therapy, has also received Breakthrough and Orphan Drug designations.

“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,” Gamida Cell CEO Julian Adams, Ph.D. said in a statement. “We are encouraged by the positive and sustained follow-up results from patients participating in the Phase III 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.”

When or if the FDA approves the BLA, the company plans to manufacture the therapy at its manufacturing facility in Israel. This is a newly constructed facility that also had the capacity to handle growth and demand for the product.

The BLA is built on data from the aforementioned Phase III trial. The primary endpoint was median time to neutrophil engraftment in patients with hematologic malignancies receiving allogeneic bone marrow transplants with omidubicel compared to standard umbilical cord blood (UCB). It demonstrated a median time to neutrophil engraftment of 12 days for patients receiving omidubicel compared to 22 days for the standard of care group. The trial also hit all secondary endpoints, including platelet engraftment, rate of infection and days alive and out of hospital. The therapy was generally well-tolerated.

“I’m quite excited about the news today. It’s another important step to bringing omidubicel to patients,” Dr. Ronit Simantov, M.D., CMO and CSO at Gamida said during a conference call Monday morning. “Patients involved in the study were ages 12-65 with high-risk hematologic malignancies. They were appropriate eligible for all-genetic stem cell transplant but had no matched donor,” she added, noting that more than 40% of the patients in the trial were ethnically diverse, a common issue in stem cell transplants.

For people with hematological cancers, such as leukemia, lymphoma and myeloma, the best treatment is an allogeneic hematopoietic stem cell transplant (HCT). However, HCT is not always available and there are also ethnic disparities in bone marrow transplant accessibility, with Black/African American patients having generally worse outcomes.

Omidubicel is cord blood that’s been taken through Gamida Cell’s manufacturing process that expands the cells using nicotinamide and uses a proprietary technology that improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process. Potentially, the cells can then be used in bone marrow transplant to repopulate the immune system of the patient who receives them.

Omidubicel requires the same matching principle that cord blood does but shows promise for ethnic minorities or people with mixed racial heritage.

In an interview with BioSpace earlier this year, Simantov said, “[Cord blood] is a wonderful source of stem cells because it doesn’t need the same level of matching that adult donors will need. If you use umbilical cord blood, you don’t need to match as exactly, so somebody who is a minority will have more likelihood of getting a match to the cord blood than a match from an adult donor registry.”