OMISIRGE

WARNING: GRAFT VERSUS HOST DISEASE, INFUSION REACTIONS, AUTOIMMUNE CYTOPENIAS, GRAFT FAILURE, and ENGRAFTMENT SYNDROME Graft-vs-Host Disease (GvHD): GvHD may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD [ see Warnings and Precautions (5.1) ]. 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 [ see Contraindications (4) , Warnings and Precautions (5.2) ]. Autoimmune cytopenias: Autoimmune cytopenias have occurred following treatment of severe aplastic anemia. Monitor blood counts prior to and after infusion. Manage cytopenias according to local institutional guidelines [see Warnings and Precautions (5.3) ]. Graft failure: Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery [ see Warnings and Precautions (5.4) ]. Engraftment syndrome: Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids [ see Warnings and Precautions (5.6) ]. WARNING: GRAFT VERSUS HOST DISEASE, INFUSION REACTIONS, AUTOIMMUNE CYTOPENIAS, GRAFT FAILURE, and ENGRAFTMENT SYNDROME See full prescribing information for complete boxed warning. Graft-vs-Host Disease (GvHD): GvHD may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD. ( 5.1 ) I nfusion 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. ( 4 , 5.2 ) Autoimmune cytopenias: Autoimmune cytopenias have occurred following treatment of severe aplastic anemia. Monitor blood counts prior to and after infusion. Manage cytopenias according to local institutional guidelines. ( 5.3 ) Graft failure: Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery. ( 5.4 ) Engraftment syndrome: Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids. ( 5.6 )

5 WARNINGS AND PRECAUTIONS Malignancies of donor origin: Monitor life-long for secondary malignancies. In the event that a secondary malignancy occurs after treatment with OMISIRGE, contact Gamida Cell at (844) 477-7478. ( 5.5 ) Transmission of serious infections: Monitor patients closely for serious infections. ( 5.7 ) Transmission of rare genetic diseases: Monitor patients for rare genetic diseases. ( 5.8 ) 5.1 Graft-versus-Host Disease Acute and chronic graft-versus-host disease (GvHD) have occurred following treatment with OMISIRGE [see Adverse Reactions (6.1) ] . 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. 5.2 Hypersensitivity and Infusion-Related Reactions Hypersensitivity and infusion-related reactions have occurred with OMISIRGE administration [see Adverse Reactions (6.1) ]. 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. 5.3 Autoimmune Cytopenias Autoimmune cytopenias (AICs) have occurred with OMISIRGE administration in patients with SAA. AIC is characterized by thrombocytopenia, anemia, and neutropenia, alone or in combination, occurring weeks to months post-transplant, often after initial hematopoietic recovery. Risk factors for post-transplant AIC include younger age, ATG-containing conditioning, underlying SAA, and delayed T cell chimerism. Monitor blood counts prior to and after OMISIRGE infusion. Manage cytopenias according to local institutional guidelines. 5.4 Graft Failure Graft failure has occurred with OMISIRGE administration [see Adverse Reactions (6.1) ] . 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. 5.5 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. 5.6 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. 5.7 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. There may be an effect on the reliability of the sterility test results if the cord blood donor was exposed to antibiotics in utero. OMISIRGE is tested for sterility, endotoxin, and mycoplasma. 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. 5.8 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.

6 ADVERSE REACTIONS Hematological malignancies: The most common adverse reactions (incidence > 20%) are infections, GvHD, and infusion and hypersensitivity reactions. ( 6.1 ) SAA: The most common adverse reactions (incidence > 20%) are infections, hyperglycemia, skin rash, febrile neutropenia, immune thrombocytopenia, acute kidney injury, acute GvHD, hypertension, hypoxia, and infusion related reactions. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Gamida Cell at (844) 477-7478 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Hematologic Malignancies The safety of OMISIRGE is based on data from Study P0501 for 52 patients transplanted with OMISIRGE and 56 patients transplanted with umbilical cord blood (UCB) [see Clinical Studies (14) ] . The median duration of follow up for the overall safety population was 14 months (range, 1-19 months). All patients received myeloablative preparative regimens and GvHD prophylaxis with tacrolimus or cyclosporin plus mycophenolate mofetil. Fatal adverse reactions occurred in 17% of patients treated with OMISIRGE, including infection (6%), acute GvHD (6%), veno-occlusive disease (VOD)/sinusoidal obstruction syndrome (SOS) (2%), thrombotic thrombocytopenic purpura (TTP)/thrombotic microangiopathy (TMA) (2%), and pulmonary hemorrhage (2%). Fatal adverse reactions occurred in 29% of subjects treated with UCB, including infection/sepsis (11%), respiratory disorders (11%), GvHD (5%), and VOD/SOS (2%). The most common non-laboratory adverse reactions occurring in ≥ 10% of patients in Study P0501 are listed in Table 2 below. The most common Grade 3-5 adverse reactions for patients treated with OMISIRGE, were pain (33%), mucosal inflammation (31%), hypertension (25%), and gastrointestinal toxicity (19%). Table 2: Adverse Reactions in ≥ 10% of Patients with Hematologic Malignancies Following Transplantation with OMISIRGE (N=52) or UCB (N=56) in Study P0501 Adverse Reaction OMISIRGE Any Grade n (%) OMISIRGE Grade 3 or Higher n (%) UCB Any Grade n (%) UCB Grade 3 or Higher n (%) Abbreviation: n: number; UCB: umbilical cord blood. 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 Fatigue includes asthenia and 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 Infections and infestations were graded according to the BMT-CTN grading system. - - - - 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 Acute Graft-versus-host disease was graded according to the Consensus Conference on Acute GvHD grading. 32 (62) 8 (15) 24 (43) 12 (21) Chronic Graft versus host disease Chronic Graft-versus-host disease was graded according to the 2014 NIH consensus criteria. 18 (35) 12 (23) 14 (25) 11 (20) Vascular disorders - - - - Hypertension 29 (56) 13 (25) 37 (66) 21 (38) Hemorrhage Hemorrhage include cystitis hemorrhagic, epistaxis, gastrointestinal hemorrhage, hemorrhage, pulmonary alveolar hemorrhage, subarachnoid hemorrhage, and upper gastrointestinal hemorrhage. 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 - - - - Arrythmia 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 disorder - - - - 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 Respiratory failure includes acute respiratory distress syndrome, acute respiratory failure, hypoxia, and 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 Renal impairment includes acute kidney injury, blood creatinine increased and renal failure. 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 Table 3 summarizes selected chemistry abnormalities by treatment arm for patients treated in Study P0501. Table 3: Chemistry Laboratory Abnormalities in ≥10% of Patients in Study P0501 OMISIRGE N = 52 UCB N = 56 Laboratory Abnormality Grade 1-4 % Grade 3-4 % Grade 1-4 % Grade 3-4 % Abbreviation: N: number; UCB: umbilical cord blood. Decreased magnesium 94 4 91 2 Increased aspartate aminotransferase 56 13 61 7 Increased alanine aminotransferase 56 13 57 9 Increased creatinine 50 4 57 2 Increased bilirubin 42 12 61 21 Increased alkaline phosphatase 42 0 54 2 Increased magnesium 15 2 29 9 Severe Aplastic Anemia The safety data described in this section reflects exposure of OMISIRGE in one clinical study (Study 17-H-0091) for the treatment of severe aplastic anemia (SAA). A total of 17 patients received a single dose of OMISIRGE with a median dose of 8.5 × 10 6 cells/kg CD34+ cells (range, 2.3- 21.4 cells/kg CD34+ cells). Three out of 17 patients received OMISIRGE with haploidentical CD34+ cells [see Clinical Studies (14) ] . All patients received a reduced intensity preparative conditioning regimen of cyclophosphamide, fludarabine, TBI and horse-ATG, and GvHD prophylaxis according to institutional guidelines. The median duration of follow-up was 25 months (range, 2-60 months). Serious adverse reactions were reported in 15 patients including infections (n=15), diarrhea (n=3), nausea/vomiting (n=4), pyrexia (n=2), hypoxia (n=2), thrombotic microangiopathy (n=1), cardiac arrest (n=1), pericarditis (n=1), colitis (n=1), febrile neutropenia (n=1), cholecystitis (n=1), portal vein thrombosis (n=1), graft-versus-host disease (n=1), weight decreased (n=1), dehydration (n=1), Guillain-Barre Syndrome (n=1), uterine hemorrhage (n=1), pleural effusion (n=1), pulmonary hemorrhage (n=1), and respiratory failure (n=1). One patient (6%) treated with OMISIRGE had a fatal adverse event. The patient engrafted but died on Day 62 from disseminated adenovirus infection. Common Terminology Criteria for Adverse Events (CTCAE) Grade 3-5 non-laboratory adverse reactions in the SAA Study with greater or equal to 15% incidence are summarized in Table 4. The most common Grade 3-5 adverse reactions for patients treated with OMISIRGE were febrile neutropenia (41%), bacterial infections (41%), hyperglycemia (41%), Epstein-Barr virus infection (29%), immune thrombocytopenia (24%) and pneumonia (24%). Table 4: Adverse Reactions in ≥15% of Patients with SAA Following Transplantation with OMISIRGE (N=17) in Study 17-H-0091 Adverse Reaction OMISIRGE Any Grade n (%) OMISIRGE Grade 3 or Higher n (%) Abbreviation: n: number. Infections and infestations - - Human herpesvirus 6 infection 16 (94) 0 BK virus infection 13 (76) 0 Bacterial infections Includes skin infection, Clostridium difficile infection, device related infections and urinary tract infections. 10 (59) 7 (41) Epstein-Barr virus infection 9 (53) 5 (29) Cytomegalovirus infection Is a composite that includes multiple related terms. 8 (47) 3 (18) Pneumonia 6 (35) 4 (24) Adenovirus infection 4 (24) 1 (6) Rhinovirus infection 4 (24) 1 (6) Sepsis 3 (18) 3 (18) Upper respiratory tract infection 3 (18) 1 (6) Metabolism and nutrition disorders - - Hyperglycemia 11 (65) 7 (41) Hypertriglyceridemia 3 (18) 2 (12) Skin and subcutaneous tissue disorders - - Skin rash 8 (47) 0 Blood and lymphatic system disorders - - Febrile neutropenia 7 (41) 7 (41) Immune thrombocytopenia 4 (24) 4 (24) Injury, poisoning and procedural complications - - Infusion related reaction Includes hypertension, headache and hypoxia. 4 (24) 2 (12) Immune system disorders - - Acute graft-versus-host disease 4 (24) 1 (6) Respiratory, thoracic and mediastinal disorders - - Hypoxia 4 (24) 3 (18) Respiratory failure Includes dyspnea, respiratory distress and respiratory failure. 3 (18) 3 (18) Vascular disorders - - Hypertension 4 (24) 3 (18) Renal and urinary disorders - - Acute kidney injury 4 (24) 2 (12) Gastrointestinal disorders - - Diarrhea 3 (18) 3 (18) Nausea 3 (18) 2 (12) Vomiting 3 (18) 2 (12) Other clinically significant adverse reactions occurring in <15% of patients include the following: Post-transplant lymphoproliferative disorder in two patients (12%), primary graft failure (defined as failure to achieve an absolute neutrophil count ≥500 cells / µl for 3 consecutive measurements on different days) in 1 patient (6%), and engraftment syndrome in 1 patient (6%). Table 5 summarizes laboratory abnormalities that worsened from baseline in ≥ 15% of patients in Study 17-H-0091. Table 5: Laboratory Abnormalities that Worsened from Baseline in ≥15% of Patients in Study 17-H-0091 (N=17) Laboratory Abnormality Grade 1-4 N (%) Grade 3-4 N (%) Abbreviation: N: number. Decreased potassium 5 (29) 0 Increased potassium 4 (24) 1 (6) Decreased phosphorous 4 (24) 0 Increased alanine aminotransferase 3 (18) 2 (12)

<table width="85%" ID="Table2"><caption>Table 2: Adverse Reactions in &#x2265; 10% of Patients with Hematologic Malignancies Following Transplantation with OMISIRGE (N=52) or UCB (N=56) in Study P0501</caption><col width="28%" align="left" valign="middle"/><col width="18%" align="center" valign="middle"/><col width="18%" align="center" valign="middle"/><col width="18%" align="center" valign="middle"/><col width="18%" align="center" valign="middle"/><thead><tr><th styleCode="Lrule Rrule">Adverse Reaction</th><th styleCode="Rrule">OMISIRGE Any Grade n (%)</th><th styleCode="Rrule">OMISIRGE Grade 3 or Higher n (%)</th><th styleCode="Rrule">UCB Any Grade n (%)</th><th styleCode="Rrule">UCB Grade 3 or Higher n (%)</th></tr></thead><tfoot><tr><td align="left" colspan="5">Abbreviation: n: number; UCB: umbilical cord blood.</td></tr></tfoot><tbody><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">General disorders and administration site conditions</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Pain</td><td styleCode="Rrule">41(79)</td><td styleCode="Rrule">17 (33)</td><td styleCode="Rrule">43 (77)</td><td styleCode="Rrule">10 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Fever</td><td styleCode="Rrule">42 (81)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">54 (96)</td><td styleCode="Rrule">6 (11)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Mucosal inflammation</td><td styleCode="Rrule">39 (75)</td><td styleCode="Rrule">16 (31)</td><td styleCode="Rrule">47 (84)</td><td styleCode="Rrule">19 (34)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Fatigue<footnote>Fatigue includes asthenia and fatigue.</footnote></td><td styleCode="Rrule">31(60)</td><td styleCode="Rrule">2 (4)</td><td styleCode="Rrule">42 (75)</td><td styleCode="Rrule">12 (21)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Edema</td><td styleCode="Rrule">24 (46)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">37 (66)</td><td styleCode="Rrule">4 (7)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Chills</td><td styleCode="Rrule">19 (37)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">32 (57)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Gastrointestinal disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Gastrointestinal toxicity</td><td styleCode="Rrule">40 (77)</td><td styleCode="Rrule">10 (19)</td><td styleCode="Rrule">48 (86)</td><td styleCode="Rrule">19 (34)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Vomiting</td><td styleCode="Rrule">33 (63)</td><td styleCode="Rrule">3 (6)</td><td styleCode="Rrule">40 (71)</td><td styleCode="Rrule">2 (4)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Dysphagia</td><td styleCode="Rrule">17 (33)</td><td styleCode="Rrule">6 (12)</td><td styleCode="Rrule">21 (38)</td><td styleCode="Rrule">7 (13)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Constipation</td><td styleCode="Rrule">12 (23)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">21 (38)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Dyspepsia</td><td styleCode="Rrule">12 (23)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">12 (21)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Abdominal distention</td><td styleCode="Rrule">10 (19)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">16 (29)</td><td styleCode="Rrule">1 (2)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Infections and infestations<footnote>Infections and infestations were graded according to the BMT-CTN grading system.</footnote></content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Viral infections</td><td styleCode="Rrule">39 (75)</td><td styleCode="Rrule">4 (8)</td><td styleCode="Rrule">45 (80)</td><td styleCode="Rrule">15 (27)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Bacterial infections</td><td styleCode="Rrule">34 (65)</td><td styleCode="Rrule">4 (8)</td><td styleCode="Rrule">45 (80)</td><td styleCode="Rrule">13 (23)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Fungal infections</td><td styleCode="Rrule">11 (21)</td><td styleCode="Rrule">3 (6)</td><td styleCode="Rrule">15 (27)</td><td styleCode="Rrule">10 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Immune System Disorder</content></td><td styleCode="Rrule"><content styleCode="bold">-</content></td><td styleCode="Rrule"><content styleCode="bold">-</content></td><td styleCode="Rrule"><content styleCode="bold">-</content></td><td styleCode="Rrule"><content styleCode="bold">-</content></td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Acute Graft versus host disease<footnote>Acute Graft-versus-host disease was graded according to the Consensus Conference on Acute GvHD grading.</footnote></td><td styleCode="Rrule">32 (62)</td><td styleCode="Rrule">8 (15)</td><td styleCode="Rrule">24 (43)</td><td styleCode="Rrule">12 (21)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Chronic Graft versus host disease<footnote>Chronic Graft-versus-host disease was graded according to the 2014 NIH consensus criteria.</footnote></td><td styleCode="Rrule">18 (35)</td><td styleCode="Rrule">12 (23)</td><td styleCode="Rrule">14 (25)</td><td styleCode="Rrule">11 (20)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Vascular disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hypertension</td><td styleCode="Rrule">29 (56)</td><td styleCode="Rrule">13 (25)</td><td styleCode="Rrule">37 (66)</td><td styleCode="Rrule">21 (38)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hemorrhage<footnote>Hemorrhage include cystitis hemorrhagic, epistaxis, gastrointestinal hemorrhage, hemorrhage, pulmonary alveolar hemorrhage, subarachnoid hemorrhage, and upper gastrointestinal hemorrhage.</footnote></td><td styleCode="Rrule">25 (48)</td><td styleCode="Rrule">6 (12)</td><td styleCode="Rrule">34 (61)</td><td styleCode="Rrule">10 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hypotension</td><td styleCode="Rrule">16 (31)</td><td styleCode="Rrule">2 (4)</td><td styleCode="Rrule">19 (34)</td><td styleCode="Rrule">5 (9)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Psychiatric disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Insomnia</td><td styleCode="Rrule">24 (46)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">26 (46)</td><td styleCode="Rrule">2 (4)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Anxiety</td><td styleCode="Rrule">15 (29)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">21(38)</td><td styleCode="Rrule">3 (5)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Depression</td><td styleCode="Rrule">13 (25)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">16 (29)</td><td styleCode="Rrule">2 (4)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Cardiac Disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Arrythmia</td><td styleCode="Rrule">24 (46)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">30 (54)</td><td styleCode="Rrule">1 (2)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Investigations</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Weight decrease/Decreased appetite</td><td styleCode="Rrule">23 (44)</td><td styleCode="Rrule">4 (8)</td><td styleCode="Rrule">22 (39)</td><td styleCode="Rrule">1 (2)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Musculoskeletal and connective tissue disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Muscular weakness</td><td styleCode="Rrule">16 (31)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">22 (39)</td><td styleCode="Rrule">2 (4)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Nervous system disorder</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Dysgeusia</td><td styleCode="Rrule">15 (29)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">9 (16)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Dizziness</td><td styleCode="Rrule">10 (19)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">13 (23)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Tremor</td><td styleCode="Rrule">8 (15)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">12 (21)</td><td styleCode="Rrule">1 (2)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Somnolence</td><td styleCode="Rrule">7 (13)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">12 (21)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Respiratory, thoracic, and mediastinal disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Cough</td><td styleCode="Rrule">14 (27)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">30 (54)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Dyspnea</td><td styleCode="Rrule">13 (25)</td><td styleCode="Rrule">4 (8)</td><td styleCode="Rrule">26 (46)</td><td styleCode="Rrule">9 (16)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Dehydration</td><td styleCode="Rrule">11 (21)</td><td styleCode="Rrule">3 (6)</td><td styleCode="Rrule">10 (18)</td><td styleCode="Rrule">2 (4)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Respiratory Failure<footnote>Respiratory failure includes acute respiratory distress syndrome, acute respiratory failure, hypoxia, and respiratory failure.</footnote></td><td styleCode="Rrule">8 (15)</td><td styleCode="Rrule">6 (12)</td><td styleCode="Rrule">26 (46)</td><td styleCode="Rrule">17 (30)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Neoplasms benign, malignant and unspecified (incl cysts and polyps)</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Disease recurrence</td><td styleCode="Rrule">11 (21)</td><td styleCode="Rrule">8 (15)</td><td styleCode="Rrule">7 (13)</td><td styleCode="Rrule">5 (14)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Renal and urinary disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Renal impairment<footnote>Renal impairment includes acute kidney injury, blood creatinine increased and renal failure.</footnote></td><td styleCode="Rrule">9 (17)</td><td styleCode="Rrule">6 (12)</td><td styleCode="Rrule">3 (5)</td><td styleCode="Rrule">3 (5)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Eye disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Dry eyes</td><td styleCode="Rrule">6 (12)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">10 (18)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Injury, poisoning and procedural complications</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Primary graft failure</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">6 (11)</td><td styleCode="Rrule">6 (11)</td></tr><tr><td styleCode="Lrule Rrule">Secondary graft failure</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">1 (2)</td><td styleCode="Rrule">0</td><td styleCode="Rrule">0</td></tr></tbody></table>

<table width="75%" ID="Table4"><caption>Table 3: Chemistry Laboratory Abnormalities in &#x2265;10% of Patients in Study P0501 </caption><col width="40%" align="left" valign="middle"/><col width="15%" align="center" valign="middle"/><col width="15%" align="center" valign="middle"/><col width="15%" align="center" valign="middle"/><col width="15%" align="center" valign="middle"/><thead><tr styleCode="Botrule"><th/><th colspan="2">OMISIRGE N = 52</th><th colspan="2">UCB N = 56</th></tr><tr><th>Laboratory Abnormality</th><th>Grade 1-4 %</th><th>Grade 3-4 %</th><th>Grade 1-4 %</th><th>Grade 3-4 %</th></tr></thead><tfoot><tr><td align="left" colspan="5">Abbreviation: N: number; UCB: umbilical cord blood.</td></tr></tfoot><tbody><tr><td>Decreased magnesium </td><td>94</td><td>4</td><td>91</td><td>2</td></tr><tr><td>Increased aspartate aminotransferase</td><td>56</td><td>13</td><td>61</td><td>7</td></tr><tr><td>Increased alanine aminotransferase</td><td>56</td><td>13</td><td>57</td><td>9</td></tr><tr><td>Increased creatinine</td><td>50</td><td>4</td><td>57</td><td>2</td></tr><tr><td>Increased bilirubin</td><td>42</td><td>12</td><td>61</td><td>21</td></tr><tr><td>Increased alkaline phosphatase</td><td>42</td><td>0</td><td>54</td><td>2</td></tr><tr><td>Increased magnesium</td><td>15</td><td>2</td><td>29</td><td>9</td></tr></tbody></table>

<table width="80%"><caption>Table 4: Adverse Reactions in &#x2265;15% of Patients with SAA Following Transplantation with OMISIRGE (N=17) in Study 17-H-0091</caption><col width="50%" align="left" valign="middle"/><col width="25%" align="center" valign="middle"/><col width="25%" align="center" valign="middle"/><thead><tr><th styleCode="Lrule Rrule">Adverse Reaction</th><th styleCode="Rrule">OMISIRGE Any Grade n (%)</th><th styleCode="Rrule">OMISIRGE Grade 3 or Higher n (%)</th></tr></thead><tfoot><tr><td align="left" colspan="3">Abbreviation: n: number.</td></tr></tfoot><tbody><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Infections and infestations</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Human herpesvirus 6 infection</td><td styleCode="Rrule">16 (94)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">BK virus infection</td><td styleCode="Rrule">13 (76)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Bacterial infections<footnote>Includes skin infection, <content styleCode="italics">Clostridium difficile</content> infection, device related infections and urinary tract infections.</footnote></td><td styleCode="Rrule">10 (59)</td><td styleCode="Rrule">7 (41)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Epstein-Barr virus infection</td><td styleCode="Rrule">9 (53)</td><td styleCode="Rrule">5 (29)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Cytomegalovirus infection<footnote ID="t4f1">Is a composite that includes multiple related terms.</footnote></td><td styleCode="Rrule">8 (47)</td><td styleCode="Rrule">3 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Pneumonia</td><td styleCode="Rrule">6 (35)</td><td styleCode="Rrule">4 (24)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Adenovirus infection</td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">1 (6)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Rhinovirus infection</td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">1 (6)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Sepsis<footnoteRef IDREF="t4f1"/></td><td styleCode="Rrule">3 (18)</td><td styleCode="Rrule">3 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Upper respiratory tract infection</td><td styleCode="Rrule">3 (18)</td><td styleCode="Rrule">1 (6)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Metabolism and nutrition disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hyperglycemia<footnoteRef IDREF="t4f1"/></td><td styleCode="Rrule">11 (65)</td><td styleCode="Rrule">7 (41)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hypertriglyceridemia</td><td styleCode="Rrule">3 (18)</td><td styleCode="Rrule">2 (12)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Skin and subcutaneous tissue disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Skin rash<footnoteRef IDREF="t4f1"/></td><td styleCode="Rrule">8 (47)</td><td styleCode="Rrule">0</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Blood and lymphatic system disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Febrile neutropenia<footnoteRef IDREF="t4f1"/></td><td styleCode="Rrule">7 (41)</td><td styleCode="Rrule">7 (41)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Immune thrombocytopenia</td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">4 (24)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Injury, poisoning and procedural complications</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Infusion related reaction<footnote>Includes hypertension, headache and hypoxia.</footnote></td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">2 (12)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Immune system disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Acute graft-versus-host disease</td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">1 (6)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Respiratory, thoracic and mediastinal disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hypoxia</td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">3 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Respiratory failure<footnote>Includes dyspnea, respiratory distress and respiratory failure.</footnote></td><td styleCode="Rrule">3 (18)</td><td styleCode="Rrule">3 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Vascular disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Hypertension</td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">3 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Renal and urinary disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Acute kidney injury</td><td styleCode="Rrule">4 (24)</td><td styleCode="Rrule">2 (12)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule"><content styleCode="bold">Gastrointestinal disorders</content></td><td styleCode="Rrule">-</td><td styleCode="Rrule">-</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Diarrhea</td><td styleCode="Rrule">3 (18)</td><td styleCode="Rrule">3 (18)</td></tr><tr styleCode="Botrule"><td styleCode="Lrule Rrule">Nausea</td><td styleCode="Rrule">3 (18)</td><td styleCode="Rrule">2 (12)</td></tr><tr><td styleCode="Lrule Rrule">Vomiting</td><td styleCode="Rrule">3 (18)</td><td styleCode="Rrule">2 (12)</td></tr></tbody></table>