Medical Drug Criteria (MDC) Medical Drug Criteria (MDC)
Policy Num: P1.002.021
Policy Name: OMISIRGE (Omidubicel-only)
Policy ID: [P1.002.021][Ac/Ar B/L M P ][0.00.00]
Last Review: September 22, 2025
Next Review: September 20, 2026
Related Policies:
| Popultation Reference No. | Populations |
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| 1 | Individuals:
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On April 17, 2023, the Food and Drug Administration approved omidubicel-onlv (Omisirge, Gamida Cell Ltd.) for use in adult 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.
Safety and efficacy were evaluated in Study P0501 (NCT02730299), an open-label, multicenter, randomized trial of omidubicel-onlv transplantation or unmanipulated cord blood (UCB) unit transplantation following myeloablative conditioning in patients with hematologic malignancies.
In total, 125 patients were randomized--62 patients to receive omidubicel-onlv and 63 to the UCB group. Fifty-two patients were transplanted with omidubicel-onlv receiving a median CD34+ cell dose of 9.0 X 106 cells/kg (range 2.1 – 47.6 X 106 cells/kg). Fifty-six patients were transplanted in the UCB arm with one or two cord units (66% received two cord units). In the 42 patients with reported post-thaw cell dose, the median CD34+ cell dose was 0.2 X 106 cells/kg (range 0.0 – 0.8 X 106 cells/kg). Multiple conditioning regimens were used, including Total Body Irradiation-based or chemotherapy-based options.
The main efficacy outcome measures were time to neutrophil recovery following transplantation and the incidence of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Grade 2/3 bacterial or Grade 3 fungal infections through Day 100 post transplantation. The median time to neutrophil recovery was 12 days for those receiving omidubicel-onlv (95% CI: 10-15 days) and 22 days in the UCB arm (95% CI: 19-25 days). Eighty-seven percent in the omidubicel-onlv arm and 83% percent in the UCB arm achieved neutrophil recovery. The incidence of BMT CTN Grade 2/3 bacterial or Grade 3 fungal infections through Day 100 post transplantation was 39% and 60%, respectively, in the two groups.
Similar to approved UCB products, the prescribing information contains a Boxed Warning for fatal or life-threatening infusion reactions, graft versus host disease (GvHD), engraftment syndrome and graft failure. Among 117 patients who received omidubicel-onlv for any disease, infusion reactions occurred in 47% of patients, acute GVHD in 58%, chronic GvHD in 35%, and graft failure in 3%.
In patients with hematologic malignancies in Study P0501, the most common Grade 3-5 adverse reactions were pain (33%), mucosal inflammation (31%), hypertension (25%), and gastrointestinal toxicity (19%).
The recommended omidubicel-onlv dose is two sequential infusions consisting of the following:
This application was granted priority review, breakthrough designation and orphan drug designation.
Blood cancers are a form of cancer caused by uncontrolled growth of cells in the blood disrupting the ability of blood cells to perform their normal functions. This abnormal cell growth often begins in the bone marrow, which is made up of stem cells that form into different types of blood cells with specific functions in the body. Blood cancers represent about 10% of all cases of cancer each year in the U.S. They can be fatal, with varying survival rates based on multiple factors including the specific type of cancer diagnosed.
- HSCT is potentially curative for patients with certain types of hematological malignancies, such as, acute lymphoblastic leukemia, acute myeloid leukemia, and myelodysplastic syndrome. Based on the origin of hematologic cells, HSCTs are classified as autologous when the patient’s own cells are used or allogeneic when the cells are from an HLA-compatible related or unrelated donor.
- The cells used in allogenic transplants can come from three sources: peripheral blood, bone marrow, and UCB. Currently, peripheral blood is the most common source for HSCT. Peripheral blood stem cells are acquired through apheresis from a donor’s peripheral vein. Bone marrow stem cells come from the liquid center of the bone and are removed via surgical procedure on a donor’s hip bone. Cord blood stem cells are collected from the umbilical cord and placenta after a baby is born. Unlike peripheral blood and bone marrow sources which require a living HLAmatched donor, UCB is donated to a cord blood bank upon birth, frozen, and stored for future use.
- For certain patients, there may be advantages to using cord blood stem cells instead of peripheral blood or bone marrow stem cells. Some potential advantages are increased availability compared to finding an HLA-matched donor; the ability to use a less closely matched HLA-donor compared to those using a peripheral or bone marrow donor; a decreased risk of developing graft-versus-host disease (GVHD); the ease of finding a donor for minority populations who traditionally are less likely to find a living HLA-matched donor; and less risk of transmission of bloodborne illnesses. Some of the disadvantages to using cord blood include a longer time to engraftment compared to bone marrow or peripheral blood due to the number of stem cells per unit of UCB being much smaller; umbilical cord blood transplant (UCBT) is a relatively new procedure in comparison to transplantation of peripheral blood or marrow stem cells; and the lack of knowledge as to how long UCB can be stored until it loses its effectiveness.
- The National Comprehensive Cancer Network (NCCN) Hematopoietic Cell Transplantation (HCT) guidelines include peripheral blood, bone marrow, or UCB as options for allogenic HSCT. Recommendations for choice of hematopoietic source are based on patient-specific factors, such as, disease type, disease stage, urgency of transplant, and patient comorbidities.
- Omisirge is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood 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.
- Human Organ Transplant Program (HOTP) of the plan is responsible for approval of all HSCTs prior to the start of treatment. Approval of the patient’s HSCT must be on file prior to determination of Omisirge’s use in any patient.
- There are no studies to support use of Omisirge following failure or after treatment with another modified allogeneic hematopoietic progenitor cell therapy derived from cord blood for the treatment of hematologic malignancies.
Hematopoietic stem-cell transplantation (HSCT) is potentially curative for patients with certain types of hematological malignancies, such as, acute lymphoblastic leukemia, acute myeloid leukemia, and myelodysplastic syndrome. Based on the origin of hematologic cells, HSCTs are classified as autologous when the patient’s own cells are used or allogeneic when the cells are from an HLA-compatible related or unrelated donor. Umbilical cord blood transplant (UCBT): Cord blood stem cells are collected from the umbilical cord and placenta after a baby is born. Unlike peripheral blood and bone marrow sources which require a living HLAmatched donor, UCB is donated to a cord blood bank upon birth, frozen, and stored for future use. Omisirge (omidubicel-onlv) is medically necessary based on the indication for use in adults and pediatric patients 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, that meet ALL of the following [documentation of medical records required]: Member is at least 12 years of age and eligible for allogenic hematopoietic stem cell transplant Member must not have a suitable matched related donor (MRD), matched unrelated donor (MUD), mismatched unrelated donor (MMUD), or haploidentical donor readily available Member must have one of the following hematologic malignancies: Acute lymphoblastic leukemia (ALL) Acute myelogenous leukemia (AML) Myelodysplastic syndrome (MDS) Chronic myeloid leukemia (CML) Lymphoma (egBurkitt's Lymphoma,High risk lymphoma,Chemotherapy-sensitive lymphomas) Other rare leukemias (egChronic myelomonocytic leukemia (CMMoL), myelodysplastic syndrome/CMMoL overlap,Biphenotypic/undifferentiated/prolymphocytic/dendritic cell leukemias and natural killer cell malignancies,Adult T-cell leukemia/lymphoma) Member has a performance score of >70% by Karnofsky or Lansky Member does not have active or uncontrolled infection Member does not have CNS disease Member does not have marked or 3+ bone marrow (BM) fibrosis Member is not pregnant or lactating Member has not received prior allogenic hematopoietic stem cell transplant Member does not have other active malignancy. When Omisirge (omidubicel-onlv) is considered medically necessary, therapy will be approved once per lifetime based on labeling: The recommended dose consists of: A Cultured Fraction (CF): a minimum of 8.0 x 108 total viable cells of which a minimum of 8.7% is CD34+ cells and a minimum of 9.2 x 107 CD34+ cells, and A Non-cultured Fraction (NF): a minimum of 4.0 x 108 total viable cells with a minimum of 2.4 x 107 CD3+ cells All other uses of Omisirge (omidubicel-onlv) are considered investigational.
OMISIRGE is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood 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.
Dose :
A single dose of OMISIRGE consists of
• a Cultured Fraction (CF): a minimum of 8.0 × 108 total viable cells of which a minimum of 8.7% is CD34+ cells and a minimum of 9.2 × 107 CD34+ cells, and
• a Non-cultured Fraction (NF): a minimum of 4.0 × 108 total viable cells with a minimum of 2.4 × 107 CD3+ cells 3 The CF and NF are supplied cryopreserved separately in two bags.
OMISIRGE requires thaw and dilution with two infusion solution (IS) bags (one IS bag for the CF, and one IS bag for the NF) prior to administration. Infusion of the NF bag should begin within 1 hour after completion of the CF infusion. For timing of dosing of each fraction, refer toPlanning prior to OMISIRGE preparation in the PI document”.
Administration:
• Do not use a leukodepleting filter (2).
• Verify patient’s identity upon receipt. Do not open the metal cassettes until time of thaw (2).
• Verify patient’s identity prior to thaw and prior to infusion (2).
• Thawing should only take place immediately prior to use (2).
• Premedicate the patient approximately 30 to 60 minutes prior to infusion
• The CF (Cultured Fraction) bag must be administered FIRST, and infusion should not exceed 2 hours from the end of dilution. Infusion of the NF (Noncultured) bag should not exceed 1 hour from the end of dilution.
• Administration of OMISIRGE should be under the supervision of a physician experienced in treatment of hematologic malignancies, in centers with expertise in hematopoietic stem cell transplants (2).
Warnings and Precautions:
Use is contraindicated in patients with known allergy to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine material. .
Benefits are determined by the group contract, member benefit booklet, and/or individual subscriber certificate in effect at the time services were rendered. Benefit products or negotiated coverages may have all or some of the services discussed in this medical policy excluded from their coverage.
Population Reference No. 1 Policy Statement
For 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. The evidence is sufficient to determine that the technology results in an improvement in the net health outcome.
| Population Reference No. 1 Policy Statement | [x ] MedicallyNecessary | [ ] Investigational |
None
1.American Academy of Pediatrics (AAP): Policy Statement The AAP is currently updating its policy on cord blood banking.
2.The American College of Obstetricians and Gynecologists (ACOG): makes the following recommendations regarding umbilical cord blood banking:
Umbilical cord blood collected from a neonate cannot be used to treat a genetic disease or malignancy in that same individual (autologous transplant) because stored cord blood contains the same genetic variant or premalignant cells that led to the condition being treated.
The routine collection and storage of umbilical cord blood with a private cord blood bank is not supported by the available evidence.
The current indications for umbilical cord blood transplantation are limited to select genetic, hematologic, and malignant disorders.
Private umbilical cord blood banking may be considered when there is knowledge of a family member with a medical condition (malignant or genetic) who could potentially benefit from cord blood transplantation.
Public umbilical cord blood banking is the recommended method of obtaining umbilical cord blood for use in transplantation, immune therapies, or other medically validated indications.
Families of all ethnicities and races should consider the societal benefit of public umbilical cord blood donation to increase the availability of matched cord blood units for people of all backgrounds.
Obstetrician–gynecologists and other obstetric care providers should be aware of state and local laws regarding umbilical cord blood banking, including the law in some states that requires physicians to inform patients about umbilical cord blood banking options.
Health care providers with a financial interest in private umbilical cord blood banking should disclose these interests, incentives, or other potential conflicts of interest.
If a patient requests information about umbilical cord blood banking, balanced and accurate information regarding the advantages and disadvantages of public and private umbilical cord blood banking should be provided.
A variety of circumstances may arise during the process of labor and delivery that may preclude adequate collection.
Umbilical cord blood collection should not compromise obstetric or neonatal care or alter routine practice of delayed umbilical cord clamping with the rare exception of medical indications for directed donation.
It is important to inform patients that the medical condition of the woman or neonate may prevent adequate umbilical cord blood collection.
3. Leukemia and Lymphoma Society: Cord blood stem cell transplants have now been given successfully to children and adult patients with malignant and nonmalignant diseases, including acute lymphoblastic leukemia (ALL); acute myeloid leukemia (AML); myelodysplastic syndromes (MDSs); chronic myeloid leukemia (CML); juvenile myelomonocytic leukemia (JMML); chronic lymphocytic leukemia (CLL); Hodgkin lymphoma (HL); non-Hodgkin lymphoma (NHL); neuroblastoma; thalassemia; severe combined immunodeficiency (SCID); Wiskott-Aldrich syndrome; metabolic diseases, such as adrenoleukodystrophy and Hurler syndrome; and severe aplastic anemia. To date, more than 35,000 cord blood stem cell transplants from unrelated donors have been performed worldwide.
Coverage and approval duration may differ for Medicare Part B members based on any applicable criteria outlined in Local Coverage Determinations (LCD) or National Coverage Determinations (NCD) as determined by Center for Medicare and Medicaid Services (CMS). See the CMS website at http://www.cms.hhs.gov/. Determination of coverage of Part B drugs is based on medically accepted indications which have supported citations included or approved for inclusion determined by CMS approved compendia.
1. Omisirge [prescribing information]. Boston, MA: Gamida Cell Inc.; January 2025.
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. 2021 Oct 21; 138 (16): 1429 - 40.
3. National Comprehensive Cancer Network. Hematopoietic Cell Transplantation (HCT) (Version 1.2025). 2025 Feb 28. Available at: https://www.nccn.org/professionals/physician_gls/pdf/hct.pdf. Accessed on March 25, 2025.
4. Leukemia and Lymphoma Society. Cord blood stem cell transplantation facts. 2016 May. Available at: https://www.lls.org/sites/default/files/2021-05/FS2_Cord_Blood_Transplantation_6_16FINAL.pdf. Accessed on April 26, 2023.
5. BCBSM and BCN medical policy
7.Horizon Medical Policy
6. U.S. Food & Drug Administraiton. FDA approves omidubicel to reduce time to neutrophil recovery and infection in patients with hematologic malignancies. Published April 17, 2023. Accessed April 25, 2023.
7. ClinicalTrails.gov. Stem Cell Transplantation with NiCord® (omidubicel) vs Standard UCB in Patients with Leukemia, Lymphoma, and MDS. NCT02730299. Available at: https://clinicaltrials.gov/ct2/show/NCT02730299https://clinicaltrials.gov/ct2/show/NCT02730299 opens a dialog window.
8. IPD Analytics. FDA Approves Gamida Cell’s Omisirge to Reduce Infection Risk in Patients with Blood Cancer. Published April 18, 2023. Accessed April 25, 2023.
9. Ballen KK, Gluckman E, Broxmeyer HE. Umbilical cord blood transplantation: the first 25 years and beyond. Blood. 2013;122(4):491-498.
10. National Cancer Institute Web site. Blood-Forming Stem Cell Transplants. www.cancer.gov/about-cancer/treatment/ types/stem-cell-transplant/stem-cell-fact-sheet. Accessed April 14, 2016.
11.The Center for Bioethics & Human Dignity Web site. Cord Blood Stem Cells: An Overview. cbhd.org/content/cordblood-stem-cells-overview. Accessed, April 14, 2016.
12.Thornley I, Eapen M, Sung L, et al. Private cord blood banking: experiences and views of pediatric hematopoietic cell transplantation physicians. Pediatrics. 2009;123(3):1011- 1017.
13.Seattle Cancer Care Alliance Web site. Improving Cord Blood Transplants. www.seattlecca.org/diseases/improvingcord-blood-transplants.cfm. Accessed, April 14, 2016.
14.US Department of Health and Human Services Food and Drug Administration Center for Biologics Evaluation and Research. Guidance for Industry: Biologics License Applications for Minimally Manipulated, Unrelated, Allogeneic Placental/ Umbilical Cord Blood Intended for Hematopoietic and Immunologic Reconstitution in Patients with Disorders Affecting the Hematopoietic System. Office of Communication, Outreach and Development (OCOD), (HFM-40), 1401 Rockville Pike, Suite 200N, Rockville, MD 20852-1448. March 2014.
15.https://www.acog.org/clinical/clinical-guidance/committee-opinion/articles/2019/03/umbilical-cord-blood-banking
16. Leukemia and Lymphoma Society https://www.lls.org/sites/default/files/2021-05/FS2_Cord_Blood_Transplantation_6_16FINAL.pdf
| Codes | Number | Description |
|---|---|---|
| HCPCS | J3590 | Unclassified biologics |
As per correct coding guidelines
| Date | Action | Description |
|---|---|---|
| September 22, 2025 | New Policy | Policy reviewed by the Pharmacy Clinical Criteria Meeting |