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Hematologic Malignancies

Efficacy and Safety

OMISIRGE demonstrated faster hematopoietic recovery with fewer infections

Primary endpoint

Patients transplanted with OMISIRGE experienced faster neutrophil recovery compared to those in the standard UCB control group1

Median time to neutrophil recovery*†

12 days

OMISIRGE (n=62)

(95% CI: 10–15 days)

87% of OMISIRGE patients achieved neutrophil recovery

22 days

Standard UCB (n=63)

(95% CI: 19–25 days)

83% of standard UCB patients achieved neutrophil recovery

10 days sooner

Absolute difference (95% CI: 6–14 days)

ITT population (n=125).

*

Time to neutrophil recovery was defined as the time from transplantation to the earliest of 3 consecutive measurements on different days with absolute neutrophil count greater than or equal to 0.5 Gi/L assessed within 42 days of follow-up.1

†

Median time to neutrophil recovery was estimated by the Kaplan-Meier estimator.1

Study design

OMISIRGE was evaluated in a global, randomized phase 3 trial across a diverse population of patients1,2

Patients enrolled (n=125)

Myeloablative

conditioning

OMISIRGE* (n=62)

Standard UCB† (n=63)

Inclusion criteria2

  • ​

Age 12–65 years

  • ​

Hematologic malignancies

  • ​

Eligible for allo-HCT

  • ​

No readily available matched donor

Endpoints2

Primary endpoint:

Time to neutrophil engraftment

Secondary endpoints:

Platelet engraftment, infection rates, days alive and out of the hospital

Tertiary endpoint:‡

NRM

Exploratory endpoints:‡

Immune reconstitution, AEs, acute GvHD, chronic GvHD, OS

*

A single dose of OMISIRGE consists of a 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 suspended in approximately 10% dimethyl sulfoxide; and a NF: a minimum of 4.0 × 108 total viable cells with a minimum of 2.4 × 107 CD3+ cells.1

†

A total of 56 patients were transplanted in the UCB arm with 1 or 2 cord units (66% received 2 cord units).1

‡

This study was not powered to detect a significant difference between groups on these endpoints. The results should be interpreted cautiously.

Who was included in the phase 3 study?

Phase 3 included patients across hematologic malignancies and risk profiles

Diagnosis at enrollment1

%

AML

48

ALL

33

MDS

7

CML

5

Lymphoma

4

Rare leukemias

3

Hematopoietic cell transplantation-specific comorbidity index2

%

Index 0

20%

Index 1-2

30%

Index ≥3

50%

Median CD34+ cell count1

9.0

× 106/kg

Range: (2.1 – 47.6) x 106

Race and ethnicity1

>40%

of patients enrolled were from historically underrepresented racial and ethnic backgrounds*

*

13% of patients identified as Hispanic or Latino

16%

Black

14%

Asian

13%

Other races or unknown

The remaining 58% of patients were White

What conditioning regimens were used in the phase 3 study?

Three myeloablative conditioning regimens were permitted: 2 TBI-containing regimens (either TBI 1350 cGy + fludarabine 160 mg/m² + thiotepa 10 mg/kg or TBI 1320 cGy + fludarabine 75 mg/m² + cyclophosphamide 120 mg/kg) and 1 chemotherapy-only regimen (thiotepa 10 mg/kg + busulfan 12.8 mg/kg + fludarabine 150 mg/m²).2

GvHD prophylaxis was provided by a calcineurin inhibitor (tacrolimus or cyclosporine) and mycophenolate mofetil starting 3 days before transplantation. Mycophenolate mofetil was continued for a minimum of 60 days and the calcineurin inhibitor for a minimum of 100 days after transplantation in the absence of toxicity or relapse.2

Explore OMISIRGE outcomes evaluated across donor sources

Retrospective CIBMTR registry analysis and OMISIRGE Expanded Access Program (EAP)

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Engraftment

How quickly did patients achieve platelet engraftment with OMISIRGE?

Secondary endpoint

Platelet engraftment achieved by Day 422

55%

OMISIRGE

vs

35%

Standard UCB

P=0.028

Median time to platelet engraftment by Day 1002*

37 days

(95% CI: 33–42)

OMISIRGE

vs

50 days

(95% CI: 42–58)

Standard UCB

P=0.023

*

Time to platelet engraftment was measured from transplantation to platelet engraftment (>20,000/μL). Platelet engraftment was defined as a platelet count >20 x 109/L without receiving a platelet transfusion in the previous 7 days.1

Infections and Immune Reconstitution

What was the incidence of bacterial and fungal infections following transplant with OMISIRGE?

Fewer invasive fungal infections through 100 days following transplant with OMISIRGE1,2

Secondary endpoint

39%

60%

OMISIRGE

Standard UCB

ITT population (n=125).2

What was the incidence of Grade 3 viral infections following transplant with OMISIRGE?

OMISIRGE demonstrated lower Grade 3 viral infections out to 1 year2*

Exploratory endpoint

10%

26%

OMISIRGE

Standard UCB

ITT population (n=125).2

*

This study was not powered to detect a significant difference between groups on this endpoint. The results should be interpreted cautiously.

How does OMISIRGE impact post-transplant immune reconstitution?

Early, rapid, and broad immune reconstitution across multiple cell lineages

OMISIRGE led to prompt multilineage immune reconstitution by 7 days post HCT3

Exploratory endpoint

OMISIRGEStandard UCB
  • Higher CD34+ progenitor cell doses correlated with rapid immune reconstitution3
  • Prompt Th cell reconstitution correlated with rapid
    neutrophil engraftment3
  • Early natural killer cell and Th cell recovery correlated with antiviral immunity post HCT3

HCT, Hematopoietic cell transplantation; Th, T helper; UCB, Umbilical cord blood.

Data from a prospectively planned optional immune reconstitution substudy instituted as part of the phase 3 study.

Post-Transplant Discharge

How did OMISIRGE impact time from transplant to hospital discharge?

Median time from transplant to discharge2*

Exploratory endpoint

27 days

OMISIRGE

35 days

Standard UCB

ITT population (n=125)2

*This study was not powered to detect a significant difference between groups on this endpoint. The results should be interpreted cautiously.

How did OMISIRGE impact time out of the hospital after transplant?

OMISIRGE was associated with more days alive and out of the hospital within the first 100 days versus standard cord blood in the first 100 days post transplant2

Secondary endpoint

61 days

OMISIRGE

48 days

Standard UCB

ITT population (n=125).2

Long-term Outcomes

What was the incidence of non-relapse mortality following transplant with OMISIRGE?

Non-relapse mortality at 210 days2*

Tertiary endpoint

11%

cumulative incidence

24%

cumulative incidence

OMISIRGE

Standard UCB

ITT, population (n=125).2

*

This study was not powered to detect a significant difference between groups on this endpoint. The results should be interpreted cautiously.

What were the longer-term overall survival outcomes with OMISIRGE?

Overall survival at 15 months2,4*

Exploratory endpoint

73%

62%

OMISIRGE

Standard UCB

ITT population (n=125).2

*

The study was not powered to see a significant difference between groups on this endpoint. The results should be interpreted cautiously.

Safety

What were the adverse events observed with OMISIRGE?

OMISIRGE has an established safety profile consistent with the expected adverse events of allo-HCT following conditioning therapy1,2

Adverse reactions in ≥10% of patients with hematologic malignancies following transplantation with OMISIRGE or standard UCB

Adverse reaction OMISIRGE
Any grade,
n (%)		 OMISIRGE
Grade 3 or higher,
n (%)		 Standard UCB
Any grade,
n (%)		 Standard UCB
Grade 3 or higher,
n (%)
General disorders and administration site conditions
Pain 41 (79) 17 (33) 43 (77) 10 (18)
Fever 42 (81) 1 (2) 54 (96) 6 (11)
Mucosal inflammation 39 (75) 16 (31) 47 (84) 19 (34)
Fatigue* 31 (60) 2 (4) 42 (75) 12 (21)
Edema 24 (46) 1 (2) 37 (66) 4 (7)
Chills 19 (37) 0 32 (57) 0
Gastrointestinal disorders
Gastrointestinal toxicity 40 (77) 10 (19) 48 (86) 19 (34)
Vomiting 33 (63) 3 (6) 40 (71) 2 (4)
Dysphagia 17 (33) 6 (12) 21 (38) 7 (13)
Constipation 12 (23) 0 21 (38) 0
Dyspepsia 12 (23) 0 12 (21) 0
Abdominal distention 10 (19) 0 16 (29) 1 (2)
Infections and infestations†
Viral infections 39 (75) 4 (8) 45 (80) 15 (27)
Bacterial infections 34 (65) 4 (8) 45 (80) 13 (23)
Fungal infections 11 (21) 3 (6) 15 (27) 10 (18)
Immune system disorder
Acute graft-versus-host disease‡ 32 (62) 8 (15) 24 (43) 12 (21)
Chronic graft-versus-host disease§ 18 (35) 12 (23) 14 (25) 11 (20)
Vascular disorders
Hypertension 29 (56) 13 (25) 37 (66) 21 (38)
HemorrhageII 25 (48) 6 (12) 34 (61) 10 (18)
Hypotension 16 (31) 2 (4) 19 (34) 5 (9)
Psychiatric disorders
Insomnia 24 (46) 1 (2) 26 (46) 2 (4)
Anxiety 15 (29) 1 (2) 21 (38) 3 (5)
Depression 13 (25) 0 16 (29) 2 (4)
Cardiac disorders
Arrhythmia 24 (46) 0 30 (54) 1 (2)
Investigations
Weight decrease/Decreased appetite 23 (44) 4 (8) 22 (39) 1 (2)
Musculoskeletal and connective tissue disorders
Muscular weakness 16 (31) 1 (2) 22 (39) 2 (4)
Nervous system disorders
Dysgeusia 15 (29) 0 9 (16) 0
Dizziness 10 (19) 0 13 (23) 0
Tremor 8 (15) 0 12 (21) 1 (2)
Somnolence 7 (13) 1 (2) 12 (21) 0
Respiratory, thoracic, and mediastinal disorders
Cough 14 (27) 0 30 (54) 0
Dyspnea 13 (25) 4 (8) 26 (46) 9 (16)
Dehydration 11 (21) 3 (6) 10 (18) 2 (4)
Respiratory failure 8 (15) 6 (12) 26 (46) 17 (30)
Neoplasms benign, malignant, and unspecified (incl cysts and polyps)
Disease recurrence 11 (21) 8 (15) 7 (13) 5 (14)
Renal and urinary disorders
Renal impairment# 9 (17) 6 (12) 3 (5) 3 (5)
Eye disorders
Dry eyes 6 (12) 0 10 (18) 0
Injury, poisoning, and procedural complications
Primary graft failure 1 (2) 1 (2) 6 (11) 6 (11)
Secondary graft failure 1 (2) 1 (2) 0 0

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*Fatigue includes asthenia and fatigue.

†Infections and infestations were graded according to the BMT-CTN grading system.

‡Acute graft-versus-host disease was graded according to the Consensus Conference on Acute GvHD grading.

§Chronic graft-versus-host disease was graded according to the 2014 NIH consensus criteria.

IIHemorrhage includes cystitis hemorrhagic, epistaxis, gastrointestinal hemorrhage, hemorrhage, pulmonary alveolar hemorrhage, subarachnoid hemorrhage, and upper gastrointestinal hemorrhage.

Respiratory failure includes acute respiratory distress syndrome, acute respiratory failure, hypoxia, and respiratory failure.

#Renal impairment includes acute kidney injury, blood creatinine increased, and renal failure.

Lab abnormalities were similar between groups

Established safety profile for OMISIRGE is consistent with the expected AEs of allo-HCT following conditioning therapy1

Chemistry laboratory abnormalities in >10% of patients (per protocol population)

Laboratory abnormality OMISIRGE, n=52 (%) Standard UCB, n=56 (%)
Grade 1-4 Grade 3-4 Grade 1-4 Grade 3-4
Magnesium decreased 94 4 91 2
Aspartate aminotransferase increased 56 13 61 7
Alanine aminotransferase increased 56 13 57 9
Creatinine increased 50 4 57 2
Bilirubin increased 42 12 61 21
Alkaline phosphatase increased 42 0 54 2
Magnesium increased 15 2 29 9
Laboratory abnormality OMISIRGE, n=52 (%)
Grade 1-4 Grade 3-4
Magnesium decreased 94 4
Aspartate aminotransferase increased 56 13
Alanine aminotransferase increased 56 13
Creatinine increased 50 4
Bilirubin increased 42 12
Alkaline phosphatase increased 42 0
Magnesium increased 15 2
Laboratory abnormality Standard UCB,
n=56 (%)
Grade 1-4 Grade 3-4
Magnesium decreased 91 2
Aspartate aminotransferase increased 61 7
Alanine aminotransferase increased 57 9
Creatinine increased 57 2
Bilirubin increased 61 21
Alkaline phosphatase increased 54 2
Magnesium increased 29 9

AE, Adverse event; ALL, Acute lymphoblastic leukemia; Allo-HCT, Allogeneic hematopoietic cell transplantation; AML, Acute myeloid leukemia; BMT-CTN, Blood & Marrow Transplant Clinical Trials; CD, Cluster of differentiation; CF, Cultured fraction; cGy, Centigray; CI, Confidence interval; CIBMTR, Center for International Blood and Marrow Transplant Research; CML, Chronic myelogenous leukemia; EQ-5D-3L, EuroQol 5 dimension 3 level; FACT-BMT, Functional Assessment of Cancer Therapy-Bone Marrow Transplantation; FACT-G, Functional Assessment of Cancer Therapy-General; GvHD, Graft-versus-host disease; HCT-CI, Hematopoietic cell transplantation-specific comorbidity index; ITT, Intent-to-treat; MDS, Myelodysplastic syndromes; NIH, National Institutes of Health; OS, Overall survival; UCB, Umbilical cord blood; TBI, Total body irradiation.

NEXT: Severe Aplastic Anemia
INDICATIONS AND USAGEIMPORTANT SAFETY INFORMATION

OMISIRGE is indicated for the treatment of 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.

IMPORTANT SAFETY INFORMATION

WARNING: GRAFT VERSUS HOST DISEASE, INFUSION REACTIONS, GRAFT FAILURE, and ENGRAFTMENT SYNDROME

  • Graft-vs-Host Disease (GvHD): GvHD may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD
  • Infusion reactions: Infusion reactions may be fatal. Monitor patients during infusion and discontinue for severe reactions. Use is contraindicated in patients with known allergy to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine material
  • Graft failure: Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery
  • Engraftment syndrome: Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids

CONTRAINDICATIONS

OMISIRGE is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine material.

WARNINGS AND PRECAUTIONS

Graft-versus-Host Disease
Acute and chronic graft versus-host disease (GvHD) have occurred following treatment with OMISIRGE. Acute GvHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Chronic GvHD manifests as skin rash, oral symptoms, ocular dryness, transaminase elevations, gastrointestinal symptoms, or serositis.

Patients treated with OMISIRGE should receive immunosuppressive drugs to decrease the risk of GvHD, and be monitored for signs and symptoms of GvHD, and treated if GvHD develops. 

Hypersensitivity and Infusion-Related Reactions
Hypersensitivity and infusion-related reactions have occurred with OMISIRGE administration.

Serious hypersensitivity reactions, including anaphylaxis, may be due to DMSO, residual gentamicin, Dextran 40, human serum albumin (HSA), and bovine material in OMISIRGE. OMISIRGE may contain residual antibiotics if the cord blood donor was exposed to antibiotics in utero. Patients with a history of allergic reactions to antibiotics should be monitored for allergic reactions following OMISIRGE administration.

Signs and symptoms of hypersensitivity reactions may include bronchospasm, wheezing, angioedema, pruritus, hives, fever, and hypotension during or after OMISIRGE infusion.

Infusion-related reactions may begin within minutes of the start of infusion of OMISIRGE, although symptoms may continue to intensify and not peak for several hours after the completion of the infusion.

Premedicate patients with antipyretics, histamine antagonists, and corticosteroids and monitor closely for signs and symptoms of hypersensitivity and infusion-related reactions. When a reaction occurs, pause the infusion and institute supportive care as needed.

Graft Failure
Graft failure has occurred with OMISIRGE administration. Primary graft failure, which may be fatal, is defined as failure to achieve an absolute neutrophil count greater than 500 per microliter blood by Day 42 after transplantation. Immunologic rejection is the primary cause of graft failure. Patients should be monitored for laboratory evidence of hematopoietic recovery.

Malignancies of Donor Origin
Malignancy of donor origin, including post-transplant lymphoproliferative disorder (PTLD), has occurred with OMISIRGE administration. PTLD manifests as a lymphoma-like disease favoring non-nodal sites. PTLD is usually fatal if not treated. The etiology is thought to be donor lymphoid cells transformed by Epstein-Barr virus (EBV). Serial monitoring of blood for EBV DNA may be warranted in patients with persistent cytopenias. A donor-cell derived myelodysplastic syndrome (MDS) has occurred with OMISIRGE administration. The natural history is presumed to be the same as that for de novo MDS. Monitor life-long for secondary malignancies. In the event that a secondary malignancy occurs, contact Gamida Cell at (844) 477-7478.

Engraftment Syndrome
Engraftment syndrome may occur because OMISIRGE is derived from umbilical cord blood. Monitor patients for unexplained fever, rash, hypoxemia, weight gain, and pulmonary infiltrates in the peri-engraftment period. Treat with corticosteroids as soon as engraftment syndrome is recognized to ameliorate symptoms. If untreated, engraftment syndrome may progress to multiorgan failure and death.

Transmission of Serious Infections
Transmission of infectious disease may occur because OMISIRGE is derived from umbilical cord blood. Disease may be caused by known or unknown infectious agents. Donors are screened for increased risk of infection with human immunodeficiency virus (HIV), human T-cell lymphotropic virus (HTLV), hepatitis B virus (HBV), hepatitis C virus (HCV), T pallidum, West Nile virus (WNV), transmissible spongiform encephalopathy (TSE) agents, vaccinia, and Zika virus [for umbilical cord blood (UCB) collected between March 2016 and 20 May 2024]. Donors are also screened for clinical evidence of sepsis and communicable disease risks associated with xenotransplantation. Maternal blood samples are tested for HIV types 1 and 2, HTLV types I and II, HBV, HCV, T pallidum, and WNV. OMISIRGE is tested for sterility, endotoxin, and mycoplasma. There may be an effect on the reliability of the sterility test results if the cord blood donor was exposed to antibiotics in utero. These measures do not totally eliminate the risk of transmitting these or other transmissible infectious diseases and disease agents. Testing of maternal and infant donor blood is also performed for evidence of donor infection due to cytomegalovirus (CMV).

Test results may be found on the container label and/or in accompanying records.

Product manufacturing includes bovine-derived reagents. While all animal-derived reagents are tested for animal viruses, bacteria, fungi, and mycoplasma before use, these measures do not eliminate the risk of transmitting these or other transmissible infectious diseases and disease agents. Final sterility test results may not be available at the time of use, but Quality Assurance (QA) will communicate any positive results from sterility testing to the physician. Report the occurrence of transmitted infection to Gamida Cell at (844) 477-7478.

Transmission of Rare Genetic Diseases
OMISIRGE may transmit rare genetic diseases involving the hematopoietic system because it is derived from umbilical cord blood. Cord blood donors have been screened to exclude donors with sickle cell anemia, and anemias due to abnormalities in hemoglobins C, D, and E. Because of the age of the donor at the time cord blood collection takes place, the ability to exclude rare genetic diseases is severely limited. Report the occurrence of transmitted rare genetic disease to Gamida Cell at (844) 477-7478. 

ADVERSE REACTIONS

The most common adverse reactions (incidence > 20%) are infections, GvHD, and infusion and hypersensitivity reactions.  

Please see full  Prescribing Information , including Boxed Warning.

References: 1. Omisirge (omidubicel-onlv). Prescribing Information. Gamida Cell Ltd.; 2026.  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;138(16):1429-1440.  3. Szabolcs P, Mazor RD, Yackoubov D, et al. Immune reconstitution profiling suggests antiviral protection after transplantation with omidubicel: a phase 3 substudy. Transplant Cell Ther. 2023;29(8):517.e1-517.e12.  4. Horwitz ME, Schiller GJ, Tsai SB, et al. Omidubicel-onlv transplantation for hematologic malignancies: results of a multicenter expanded access program. Transplant Cell Ther. 2025;31(7):436-447.