FDA Approves Gamida Cell’s Omisirge for Planned Umbilical Cord Blood Transplantation

The FDA has approved omidubicel-onlv (Omisirge; Gamida Cell Ltd) for use in individuals aged 12 years and older with blood cancer planned for umbilical cord blood transplantation, following a myeloablative conditioning regimen, including radiation and chemotherapy.

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Omidubicel-onlv is a modified allogenic cord blood-based cell therapy that is intended to quicken the recovery of neutrophils, a subset of white blood cells, and reduce the risk of infection.

“[This] approval is an important advance in cell therapy treatment in patients with blood cancers,” Peter Marks, MD, PhD, director of the Center for Biologics Evaluation and Research at the FDA, said in a statement.

“Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation,” he said. “This approval reflects the FDA’s continued commitment to supporting development of innovative therapies for life-threatening cancers.”

Omidubicel-onlv includes human allogeneic stem cells from umbilical cord blood that are cultured and processed with nicotinamide, a formation of vitamin B3, and is designed to be used as a single, intravenous dose. The doses are patient-specific and contain healthy stem cells from pre-screened donors.

The treatment’s effectiveness and safety were supported by the results of a multicenter, randomized study (NCT02730299) comparing the transplantation of the drug to the transplantation of umbilical cord blood. The study included 125 individuals, aged 12 to 65 years with confirmed blood cancers.

The efficacy of omidubicel-onlv was based on the amount of time needed for individuals to recover white blood cells, which offer protection from infection, and the incidence of infections after transplantation.

Approximately 87% of individuals who were given omidubicel-onlv achieved neutrophil recovery with a median of 12 days following treatment compared with 83% who received umbilical cord blood transplantation and had a 22-day median for recovery, investigators reported.

Furthermore, bacterial or fungal infections, by 100 days post-transplantation, were seen in approximately 39% of individuals who received the drug compared with 60% who received umbilical cord blood.

However, treatment with omidubicel-onlv may cause severe adverse effects (AEs), which should be considered when assessing benefits and risks.

The AEs are similar to approved umbilical cord products, including engraftment syndrome, characterized by a non-infection fever and rash; graft failure, which occurs when new cells do not produce platelets and red and white blood cells; graft versus host disease (GvHD), which occurs when donor bone marrow or stem cells attack the individual; and infusion reaction.

The most common AEs with omidubicel-onlv included GvHD, infection, and infusion reaction. Individuals who receive the drug should also be monitored for signs and symptoms of engraftment syndrome; graft failure; GvHD; infusion reaction; secondary malignancies; which can spread from the original cancer site or emerge after treatment; and transmission of rare genetic diseases or serious infections from the donor cells.

The application previously received priority review, breakthrough therapy, and orphan designation.

Reference

FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplantation. US Food and Drug Administration. April 17, 2023. Accessed April 18, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell?utm_medium=email&utm_source=govdelivery