Omidubicel

Identification

Summary

Omidubicel is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood indicated for use in patients with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning.

Generic Name
Omidubicel
DrugBank Accession Number
DB17132
Background

Omidubicel is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood used as an allogeneic stem cell donor source. Umbilical blood cord cells are expanded ex-vivo and nicotinamide (a form of vitamin B3) is used as an active agent in the culture system to inhibit the differentiation and increase the functionality of hematopoietic stem and progenitor cells.1,2,5 This type of cell therapy benefits patients that require stem cell transplantation as a treatment for blood cancers for whom a related or unrelated donor cannot be found due to the requirement of high-grade human leukocyte antigen (HLA)-matching. In this group of patients, umbilical cord blood can be used as an alternative cell source since there is a lower risk of graft failure or graft-versus-host disease, even with low-grade HLA-matching.3

A pooled analysis of five prospective clinical trials found that the use of omidubicel led to stable trilineage hematopoiesis, immune competence, and graft durability in extended follow-up. In addition, the fact that 39% of the study's cohort were non-White patients highlights the importance of this type of treatment in key demographics with low donor availability.4 In April 2023, the FDA approved the use of omidubicel in adults and pediatric patients 12 years and older with blood cancers planned for umbilical cord blood transplantation following a myeloablative conditioning regimen. It is administered as a single intravenous dose specific to each patient.5,6

Type
Biotech
Groups
Approved, Investigational
Biologic Classification
Cell transplant therapies
Other cell transplant therapies
Synonyms
  • Allogeneic, ex-vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells - cultured fraction (CF)
  • NiCord
  • Omidubicel
  • Omidubicel-onlv

Pharmacology

Indication

Omidubicel is indicated for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.5

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Associated Therapies
Contraindications & Blackbox Warnings
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Pharmacodynamics

Omidubicel is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood. As early as one-week post-transplantation, the use of omidubicel results in rapid and broad immune reconstitution of dendritic cells, monocytes, natural killer (NK) cells, CD4+ T cells and CD8+ T cells. In the cultured fraction (CF) of omidubicel, there is a positive linear correlation between the CD34+ cell content and the reconstitution of NK and T-cells. The total CD34+ cell counts and the omidubicel dose also strongly correlate with the kinetics of neutrophil recovery. For instance, higher CD34+ cell doses lead to lower days to neutrophil recovery.5

The use of omidubicel can lead to severe side effects such as graft versus host disease, engraftment syndrome and graft failure, and is associated with a higher incidence of infections and infusion reactions.5,6

Mechanism of action

Omidubicel is a cryopreserved nicotinamide-modified allogeneic hematopoietic progenitor cell therapy manufactured from patient-specific umbilical cord blood (UCB) and processed in a way that enhances the functionality of hematopoietic progenitor cells (HPCs). A single banked UCB unit is separated into 2 cell fractions by CD133 selection, forming a CD133+ or cultured fraction (CF) and a CD133− or non-cultured fraction (NF). The CD133+ or CF is expanded using nicotinamide, a small molecule that inhibits differentiation, increases the frequency of CD34+ HPCs and decreases the frequency of lineage-committed progenitor cells.1,2,5 The use of nicotinamide overcomes the induction of accelerated proliferation, differentiation, cellular stress and signaling pathways that are typically activated when HPCs are removed from their natural environment. This allows HPCs derived from UCB and cultured ex-vivo to preserve their stemness, increasing their capacity for bone marrow migration, rapid engraftment and multi-lineage immune reconstitution.1,5

The use of this cell therapy is beneficial in patients with hematologic malignancies that do not have HLA-matched related donors, as it allows them to restore their ability to produce blood and immune cells.3,5

Absorption

There is limited pharmacokinetic information on omidubicel.

Volume of distribution

There is limited pharmacokinetic information on omidubicel.

Protein binding

There is limited pharmacokinetic information on omidubicel.

Metabolism

There is limited pharmacokinetic information on omidubicel.

Route of elimination

There is limited pharmacokinetic information on omidubicel.

Half-life

There is limited pharmacokinetic information on omidubicel.

Clearance

There is limited pharmacokinetic information on omidubicel.

Adverse Effects
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Toxicity

Toxicity information regarding omidubicel is not readily available. Patients experiencing an overdose are at an increased risk of severe adverse effects such as severe infusion reactions and graft-versus-host disease (GVHD).5 Symptomatic and supportive measures are recommended.

Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
No interactions found.

Products

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International/Other Brands
Omisirge (Gamida Cell Inc.)

Categories

Drug Categories
Classification
Not classified
Affected organisms
  • Humans and other mammals

Chemical Identifiers

UNII
ET4JC4S66E
CAS number
Not Available

References

Synthesis Reference

Peled, T., et al. (2011). Expansion of renewable stem cell populations (U.S. Patent No. 7,955,852 B2). U.S. Patent and Trademark Office. https://patentimages.storage.googleapis.com/55/39/b0/98823954f3bdba/US7955852.pdf

General References
  1. Horwitz ME, Chao NJ, Rizzieri DA, Long GD, Sullivan KM, Gasparetto C, Chute JP, Morris A, McDonald C, Waters-Pick B, Stiff P, Wease S, Peled A, Snyder D, Cohen EG, Shoham H, Landau E, Friend E, Peleg I, Aschengrau D, Yackoubov D, Kurtzberg J, Peled T: Umbilical cord blood expansion with nicotinamide provides long-term multilineage engraftment. J Clin Invest. 2014 Jul;124(7):3121-8. doi: 10.1172/JCI74556. Epub 2014 Jun 9. [Article]
  2. Horwitz ME, Stiff PJ, Cutler C, Brunstein C, Hanna R, Maziarz RT, Rezvani AR, Karris NA, McGuirk J, Valcarcel D, Schiller GJ, Lindemans CA, Hwang WYK, Koh LP, Keating A, Khaled Y, Hamerschlak N, Frankfurt O, Peled T, Segalovich I, Blackwell B, Wease S, Freedman LS, Galamidi-Cohen E, Sanz G: Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021 Oct 21;138(16):1429-1440. doi: 10.1182/blood.2021011719. [Article]
  3. de Koning C, Tao W, Lacna A, van Veghel K, Horwitz ME, Sanz G, Jagasia MH, Wagner JE, Stiff PJ, Hanna R, Cilloni D, Valcarcel D, Peled T, Galamidi Cohen E, Goshen U, Pandit A, Lindemans CA, Jan Boelens J, Nierkens S: Lymphoid and myeloid immune cell reconstitution after nicotinamide-expanded cord blood transplantation. Bone Marrow Transplant. 2021 Nov;56(11):2826-2833. doi: 10.1038/s41409-021-01417-4. Epub 2021 Jul 26. [Article]
  4. Lin C, Schwarzbach A, Sanz J, Montesinos P, Stiff P, Parikh S, Brunstein C, Cutler C, Lindemans CA, Hanna R, Koh LP, Jagasia MH, Valcarcel D, Maziarz RT, Keating AK, Hwang WYK, Rezvani AR, Karras NA, Fernandes JF, Rocha V, Badell I, Ram R, Schiller GJ, Volodin L, Walters MC, Hamerschlak N, Cilloni D, Frankfurt O, McGuirk JP, Kurtzberg J, Sanz G, Simantov R, Horwitz ME: Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials. Transplant Cell Ther. 2023 Feb 10:S2666-6367(23)01071-0. doi: 10.1016/j.jtct.2023.01.031. [Article]
  5. FDA Approved Drug Products: OMISIRGE (omidubicel-onlv) suspension for infusion for intravenous use (April 2023) [Link]
  6. US Food & Drug Administration: FDA Approves Cell Therapy for Patients with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation [Link]
RxNav
2637354
Wikipedia
Omidubicel

Clinical Trials

Clinical Trials

Pharmacoeconomics

Manufacturers
Not Available
Packagers
Not Available
Dosage Forms
Not Available
Prices
Not Available
Patents
Not Available

Properties

State
Liquid
Experimental Properties
Not Available

Drug created at November 18, 2022 14:43 / Updated at April 28, 2023 11:47