On September 27, 2023 Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working to turn cells into powerful therapeutics, reported that the first patient has received a stem cell transplant with Omisirge (omidubicel-onlv) (Press release, Gamida Cell, SEP 27, 2023, View Source [SID1234635486]).
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"This is a significant milestone for Gamida Cell, advancing our mission of delivering potentially curative therapies to patients with cancer," said Abbey Jenkins, President and Chief Executive Officer of Gamida Cell. "This patient will be the first of many who have new hope for a cure thanks to the availability of Omisirge as a new stem cell transplant donor source. This is why we do the work that we do – to make a difference for people with cancer."
Omisirge was approved by the U.S. FDA in April 2023 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.
Gamida Cell has already exceeded its 2023 launch goals, with 15 transplant centers onboarded across the United States and confirmed coverage with payers that cover 90% of commercial lives. Gamida Cell is actively engaged with more than 90% of the top 70 transplant centers, which conduct approximately 80% of transplants. An increasing number of patients are being enrolled in Gamida Cell Assist, which signals a transplanter’s intent to use Omisirge as the donor source.
"The launch of Omisirge is progressing very well in terms of payer coverage, transplant center onboarding and transplanter interest in using Omisirge as a donor source," said Michele Korfin, Chief Operating and Chief Commercial Officer of Gamida Cell. "We recognize the importance of making sure eligible patients can access Omisirge. To appropriately manage our cash, we launched with a limited investment and field footprint. The positive launch progress and strong interest from transplant centers now warrant expanding that investment and the team from four to eight account managers by the start of 2024. We are encouraged by transplanter feedback that Omisirge may both increase the number of patients able to access an appropriate donor source and address some limitations of other donor sources."
Approximately 8,000 stem cell transplants are performed in the U.S. each year in patients with hematologic malignancies1 and another 1,700 patients are estimated to be eligible for transplant but unable to find a donor.2 The ability to find a donor is historically more challenging for racially and ethnically diverse populations than for patients who are white.3 Gamida Cell market analyses indicate that Omisirge has the ability to capture approximately 20% of allogeneic stem cell transplant market share by ~2028.
Omisirge Indication
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.
Important Safety Information for Omisirge
BOXED WARNING: INFUSION REACTIONS, GRAFT VERSUS HOST DISEASE, ENGRAFTMENT SYNDROME, AND GRAFT FAILURE
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-versus-Host Disease may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD.
Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids.
Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery.
Contraindications
OMISIRGE is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine products.
Warnings and Precautions
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of OMISIRGE. Reactions include bronchospasm, wheezing, angioedema, pruritis and hives. 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.
Infusion Reactions
Infusion reactions occurred following OMISIRGE infusion, including hypertension, mucosal inflammation, dysphagia, dyspnea, vomiting, and gastrointestinal toxicity. Premedication with antipyretics, histamine antagonists, and corticosteroids may reduce the incidence and intensity of infusion reactions. In patients transplanted with OMISIRGE in clinical trials, 47% (55/117) patients had an infusion reaction of any severity. Grade 3-4 infusion reactions were reported in 15% (18/117) patients. Infusion 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. Monitor patients for signs and symptoms of infusion reactions during and after OMISIRGE administration. When a reaction occurs, pause the infusion and institute supportive care as needed.
Graft-versus-Host Disease
Acute and chronic GvHD, including life-threatening and fatal cases, occurred following treatment with OMISIRGE. In patients transplanted with OMISIRGE Grade II-IV acute GvHD was reported in 58% (68/117). Grade III- IV acute GvHD was reported in 17% (20/117). Chronic GvHD occurred in 35% (41/117) of patients. Acute GvHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Patients treated with OMISIRGE should receive immunosuppressive drugs to decrease the risk of GvHD, be monitored for signs and symptoms of GvHD, and treated if GvHD develops.
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.
Graft Failure
Primary graft failure occurred in 3% (4/117) of patients in OMISIRGE clinical trials. 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. Monitor patients for laboratory evidence of hematopoietic recovery.
Malignancies of Donor Origin
Two patients treated with OMISIRGE developed post-transplant lymphoproliferative disorder (PTLD) in the second-year post-transplant. 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. One patient treated with OMISIRGE developed a donor-cell derived myelodysplastic syndrome (MDS) during the fourth-year post-transplant. The natural history is presumed to be the same as that for de novo MDS. Monitor life-long for secondary malignancies. If a secondary malignancy occurs, contact Gamida Cell at (844) 477-7478.
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, clinical evidence of sepsis, and communicable disease risks associated with xenotransplantation. Maternal and infant donor blood is tested for evidence of donor infection. See full Prescribing Information, Warnings and Precautions, Transmission of Serious Infections for list of testing performed. 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. Product manufacturing includes bovine-derived reagents. 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. Test results may be found on the container label and/or in accompanying records. If final sterility results are not available at the time of use, Quality Assurance 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.
ADVERSE REACTIONS
The most common adverse reactions (incidence > 20%) are infections, GvHD, and infusion reaction.
Please see full Prescribing Information, including Boxed Warning.