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April 25, 2023April 17, 2023 - the FDA approved Gamida Cell’s Omisirge (omidubicel-onlv), a nicotinamide-modified allogeneic cord blood–based hematopoietic progenitor cell therapy to reduce the time to neutrophil recovery and the incidence of infection in adult and pediatric patients 12 years of age and older with blood cancers, who are planned for umbilical cord blood transplantation (UCBT) following a myeloablative conditioning regimen.
Omisirge is composed of human allogeneic hematopoietic stem cells from umbilical cord blood (UCB) that are processed and cultured with nicotinamide. Administered intravenously, Omisirge consists of a one-time, patient-specific dose of at least 12 × 10 8 live cells, which include CD34+ and CD3+ cells. While a study conducted in a Phase 3 open-label multicenter, randomized trial compared Omisirge to standard UCB, the median time to neutrophil recovery was shortened from 22 days to 12 days,
The approval was supported by results of Study P0501 (NCT02730299), a Phase 3 open-label, multicenter, randomized trial that compared Omisirge to standard (unmanipulated) UCB in 125 patients between 12 and 65 years of age with blood cancers. In the trial, 87% of patients who received Omisirge achieved neutrophil recovery, compared to 83% of patients in the standard UCB group. The median time to neutrophil recovery was 12 days in the Omisirge group and 22 days in the standard UCB group. Patients in the Omisirge group also showed significant improvements in platelet recovery and time out of hospital compared with those in the standard UCB group. However, Omisirge did not significantly improve nonrelapse mortality, disease relapse, disease-free survival, overall survival, or graft-versus- host disease (GVHD) in comparison to standard UCB.
Similar to all FDA-approved umbilical cord products, the label for Omisirge carries a Boxed Warning for fatal infusion reactions, GVHD, engraftment syndrome, and graft failure.
By 2027, Omisirge has the potential to treat approximately 2000–2500 patients per year in the United States with hematologic malignancies who are eligible for transplant but unable to find a donor source, thus filling an unmet need for this population. The Biologics License Application (BLA) for Omisirge received Priority Review, Breakthrough Therapy, and Orphan Drug designations.
Omisirge has a wholesale acquisition cost (WAC) of $338,000 for the one-time treatment and is now available to transplant centers. There is a 30-day turnaround time from the start of manufacturing.
IPD Analytics recommends the use of management tools such as prior authorization to ensure appropriate utilization of Omisirge.