The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for omidubicel for the treatment of patients with hematologic malignancies in need of an allogeneic hematopoietic stem cell transplant.

Omidubicel is a first-in-class, advanced NAM (nicotinamide)-enabled stem cell therapy candidate. The application is supported by data from a multicenter, randomized, phase 3 trial (ClinicalTrials.gov Identifier: NCT02730299), which evaluated the efficacy of omidubicel in 125 patients, 13 to 65 years of age with hematologic malignancies (eg, acute lymphoblastic leukemia, acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma), who were eligible for allogeneic transplantation.

Patients were randomly assigned to receive omidubicel or standard umbilical cord blood transplantation (UCBT). The primary endpoint was time to neutrophil engraftment.

Results showed that the median time to neutrophil engraftment was 12 days (95% CI, 10-15) for the omidubicel arm and 22 days (95% CI, 19-25) for the standard UCBT arm (P <.001). Treatment with omidubicel also met all secondary endpoints, including faster platelet recovery (55% vs 35% by 42 days; P =.028), a lower incidence of infections (37% vs 57%; P =.027), and reduced hospitalization during the first 100 days after transplant (median, 61 vs 48 days; P =.005) compared with UCBT. The treatment was found to be well tolerated.

A Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2023 has been set for the application.

“We are encouraged by the positive and sustained follow-up results from patients participating in the phase 3 trial of omidubicel, including a positive overall survival trend 1-year out from treatment,” said Julian Adams, PhD, CEO of Gamida Cell. “These results provide promising rationale that, if approved, omidubicel could become a treatment of choice for patients in need of an allo-HSCT transplant.”

References

  1. Gamida Cell announces FDA acceptance of Biologics License Application for omidubicel with Priority Review. News release. Gamida Cell Ltd. Accessed August 1, 2022. https://www.businesswire.com/news/home/20220801005265/en/Gamida-Cell-Announces-FDA-Acceptance-of-Biologics-License-Application-for-Omidubicel-with-Priority-Review
  2. Horwitz ME, Stiff PJ, Cutler C, et al. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. Published online October 21, 2021. doi:10.1182/blood.2021011719