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Drug overview for ZYNTEGLO (betibeglogene autotemcel):
Generic name: betibeglogene autotemcel (BE-ti-BEG-lo-jeen AW-to-TEM-sel)
Drug class: Hematopoietic Agents - Hematopoietic Gene Therapy Agents
Therapeutic class: Hematological Agents
Betibeglogene autotemcel is an autologous hematopoietic stem cell (HSC)-based gene therapy.
No enhanced Uses information available for this drug.
Generic name: betibeglogene autotemcel (BE-ti-BEG-lo-jeen AW-to-TEM-sel)
Drug class: Hematopoietic Agents - Hematopoietic Gene Therapy Agents
Therapeutic class: Hematological Agents
Betibeglogene autotemcel is an autologous hematopoietic stem cell (HSC)-based gene therapy.
No enhanced Uses information available for this drug.
DRUG IMAGES
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The following indications for ZYNTEGLO (betibeglogene autotemcel) have been approved by the FDA:
Indications:
Anemia in beta thalassemia
Professional Synonyms:
None.
Indications:
Anemia in beta thalassemia
Professional Synonyms:
None.
The following dosing information is available for ZYNTEGLO (betibeglogene autotemcel):
It isessential that the manufacturer's labeling be consulted for more detailed information on dosage and administration of this drug. Dosage summary:
For autologous use only. For IV use only.
*Patients are required to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for betibeglogene autotemcel manufacturing.
*Full myeloablative conditioning must be administered before infusion of betibeglogene autotemcel.
*Prophylaxis for hepatic veno-occlusive disease (VOD) is recommended. Prophylaxis for seizures should be considered.
*Verify that the patient's identity matches the unique patient identification information on the betibeglogene autotemcel infusion bag(s) prior to infusion.
*Do not sample, alter, or irradiate betibeglogene autotemcel.
*Do not use an in-line blood filter or an infusion pump.
*Administer each infusion bag of betibeglogene autotemcel via IV infusion over a period of less than 30 minutes.
*See Full Prescribing Information for additional details and preparation and administration.
Dosing of betibeglogene autotemcel is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight.
The minimum recommended dose is 5.0 x 106 CD34+ cells/kg in pediatric patients older than 4 years of age. Safety and efficacy have not been established in pediatric patients younger than 4 years of age.
Dosing of betibeglogene autotemcel is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight.
The minimum recommended dose is 5.0 x 106 CD34+ cells/kg.
For autologous use only. For IV use only.
*Patients are required to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for betibeglogene autotemcel manufacturing.
*Full myeloablative conditioning must be administered before infusion of betibeglogene autotemcel.
*Prophylaxis for hepatic veno-occlusive disease (VOD) is recommended. Prophylaxis for seizures should be considered.
*Verify that the patient's identity matches the unique patient identification information on the betibeglogene autotemcel infusion bag(s) prior to infusion.
*Do not sample, alter, or irradiate betibeglogene autotemcel.
*Do not use an in-line blood filter or an infusion pump.
*Administer each infusion bag of betibeglogene autotemcel via IV infusion over a period of less than 30 minutes.
*See Full Prescribing Information for additional details and preparation and administration.
Dosing of betibeglogene autotemcel is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight.
The minimum recommended dose is 5.0 x 106 CD34+ cells/kg in pediatric patients older than 4 years of age. Safety and efficacy have not been established in pediatric patients younger than 4 years of age.
Dosing of betibeglogene autotemcel is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight.
The minimum recommended dose is 5.0 x 106 CD34+ cells/kg.
No enhanced Administration information available for this drug.
No dosing information available.
No generic dosing information available.
The following drug interaction information is available for ZYNTEGLO (betibeglogene autotemcel):
There are 0 contraindications.
There are 2 severe interactions.
These drug interactions can produce serious consequences in most patients. Actions required for severe interactions include, but are not limited to, discontinuing one or both agents, adjusting dosage, altering administration scheduling, and providing additional patient monitoring. Review the full interaction monograph for more information.
| Drug Interaction | Drug Names |
|---|---|
| Betibeglogene Autotemcel/Anti-Retrovirals; Hydroxyurea SEVERITY LEVEL: 2-Severe Interaction: Action is required to reduce the risk of severe adverse interaction. MECHANISM OF ACTION: Betibeglogene autotemcel is prepared from apheresed cells that are transduced with a replication defective, self-inactivating lentiviral vector. Antiretrovirals may interfere with the manufacturing of apheresed cells. Hydroxyurea may interfere with hematopoietic stem cell (HSC) mobilization of CD34+ cells.(1) CLINICAL EFFECTS: Use of hydroxyurea before mobilization may result in unsuccessful stem cell mobilization. Use of antiretrovirals before mobilization and apheresis may interfere with the production of betibeglogene autotemcel. PREDISPOSING FACTORS: None determined. PATIENT MANAGEMENT: Discontinue antiretrovirals and hydroxyurea for at least one month prior to mobilization and until all cycles of apheresis are completed. If a patient requires antiretrovirals for HIV prophylaxis, then confirm a negative HIV test before beginning mobilization and apheresis of CD34+ cells. DISCUSSION: Antiretroviral medications and hydroxyurea may interfere with the manufacturing of betibeglogene autotemcel therapy.(1) |
ABACAVIR, ABACAVIR-LAMIVUDINE, APRETUDE, APTIVUS, ATAZANAVIR SULFATE, BIKTARVY, CABENUVA, CABOTEGRAVIR ER (CABENUVA), CIMDUO, COMPLERA, DARUNAVIR, DELSTRIGO, DESCOVY, DOVATO, DROXIA, EDURANT, EDURANT PED, EFAVIRENZ, EFAVIRENZ-EMTRIC-TENOFOV DISOP, EFAVIRENZ-LAMIVU-TENOFOV DISOP, EMTRICITABINE, EMTRICITABINE-RILPIVIRNE-TENOF, EMTRICITABINE-TENOFOVIR DISOP, EMTRIVA, EPIVIR, ETRAVIRINE, EVOTAZ, FOSAMPRENAVIR CALCIUM, FUZEON, GENVOYA, HYDREA, HYDROXYUREA, INTELENCE, ISENTRESS, ISENTRESS HD, JULUCA, KALETRA, LAMIVUDINE, LAMIVUDINE HBV, LAMIVUDINE-ZIDOVUDINE, LOPINAVIR-RITONAVIR, MARAVIROC, NEVIRAPINE, NEVIRAPINE ER, NORVIR, ODEFSEY, PAXLOVID, PIFELTRO, PREZCOBIX, PREZISTA, RETROVIR, REYATAZ, RILPIVIRINE ER (CABENUVA), RITONAVIR, RUKOBIA, SELZENTRY, SIKLOS, STRIBILD, SYMFI, SYMTUZA, TENOFOVIR DISOPROXIL FUMARATE, TIVICAY, TIVICAY PD, TRIUMEQ, TRIUMEQ PD, TROGARZO, TRUVADA, TYBOST, VEMLIDY, VIRACEPT, VIREAD, VOCABRIA, XROMI, ZIAGEN, ZIDOVUDINE |
| Betibeglogene Autotemcel/Myelosuppressive Iron Chelators SEVERITY LEVEL: 2-Severe Interaction: Action is required to reduce the risk of severe adverse interaction. MECHANISM OF ACTION: Treatment with betibeglogene autotemcel requires hematopoietic stem cell (HSC) mobilization which can suppress the immune system. CLINICAL EFFECTS: Myelosuppressive iron chelators may result in increased risk of serious infections. PREDISPOSING FACTORS: None determined. PATIENT MANAGEMENT: Avoid myelosuppressive iron chelators for six months after betibeglogene autotemcel infusion. If iron chelation is needed, consider administration of non-myelosuppressive iron chelators.(1) DISCUSSION: In clinical trials, 100% of 41 patients experienced Grade 3 or 4 neutropenia from Day 1 to Month 24.(1) |
DEFERIPRONE, DEFERIPRONE (3 TIMES A DAY), FERRIPROX, FERRIPROX (2 TIMES A DAY), FERRIPROX (3 TIMES A DAY) |
There are 0 moderate interactions.
The following contraindication information is available for ZYNTEGLO (betibeglogene autotemcel):
Drug contraindication overview.
None.
None.
There are 1 contraindications.
Absolute contraindication.
| Contraindication List |
|---|
| Lactation |
There are 0 severe contraindications.
There are 0 moderate contraindications.
The following adverse reaction information is available for ZYNTEGLO (betibeglogene autotemcel):
Adverse reaction overview.
The most common non-laboratory adverse reactions (incidence >= 20%) were mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nose bleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus (itch). The most common Grade 3 or 4 laboratory abnormalities (> 50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.
The most common non-laboratory adverse reactions (incidence >= 20%) were mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nose bleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus (itch). The most common Grade 3 or 4 laboratory abnormalities (> 50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.
There are 19 severe adverse reactions.
| More Frequent | Less Frequent |
|---|---|
|
Anemia Leukopenia Lymphopenia Neutropenic disorder Thrombocytopenic disorder |
Chronic heart failure Dyspnea Hepatic veno-occlusive disease Hyperbilirubinemia Hypertension Hypokalemia Hyponatremia Hypophosphatemia Hypoxia Increased alanine transaminase Pneumonia Sepsis Viral infection |
| Rare/Very Rare |
|---|
|
Malignancy |
There are 22 less severe adverse reactions.
| More Frequent | Less Frequent |
|---|---|
|
Acute abdominal pain Alopecia Constipation Cough Epistaxis Fever Headache disorder Musculoskeletal pain Nausea Pruritus of skin Stomatitis Vomiting |
Anorexia Diarrhea Dyschromia Fatigue Hyperglycemia Pain in oropharynx Pharyngitis Rhinitis Skin rash Upper respiratory infection |
| Rare/Very Rare |
|---|
| None. |
The following precautions are available for ZYNTEGLO (betibeglogene autotemcel):
The safety and efficacy of betibeglogene autotemcel have been established in pediatric patients with beta-thalassemia requiring regular transfusions. Use of betibeglogene autotemcel is supported by two Phase 3 studies that included 27 pediatric patients in the following age groups: 16 children (less than 12 years of age) and 11 adolescents (12 years to less than 18 years of age). No differences in efficacy or clinical safety were observed between the adult and pediatric subgroups.
Engraftment times were longer in pediatric patients, but not associated with increases in infections or bleeding events. The median (min, max) time to neutrophil engraftment for patients less than 18 years was 26 (16, 39) days versus 21 (13, 27) days for patients 18 years or older. The median (min, max) time to platelet engraftment for patients less than 18 years was 50 (20, 94) days versus 43 (21, 58) days for patients 18 years or older.
Longer engraftment time was associated with intact spleens. The safety and efficacy of betibeglogene autotemcel in children less than 4 years of age have not been established. No data are available.
Contraindicated
Severe Precaution
Management or Monitoring Precaution
Engraftment times were longer in pediatric patients, but not associated with increases in infections or bleeding events. The median (min, max) time to neutrophil engraftment for patients less than 18 years was 26 (16, 39) days versus 21 (13, 27) days for patients 18 years or older. The median (min, max) time to platelet engraftment for patients less than 18 years was 50 (20, 94) days versus 43 (21, 58) days for patients 18 years or older.
Longer engraftment time was associated with intact spleens. The safety and efficacy of betibeglogene autotemcel in children less than 4 years of age have not been established. No data are available.
Contraindicated
| None |
Severe Precaution
| None |
Management or Monitoring Precaution
| None |
There are no available data with betibeglogene autotemcel administration in pregnant women. Consider the risks associated with myeloablative conditioning agents on pregnancy and fertility. No reproductive and developmental toxicity studies in animals have been conducted with betibeglogene autotemcel to assess whether it can cause fetal harm when administered to a pregnant woman.
It is not known whether betibeglogene autotemcel has the potential to be transferred to the fetus. Therefore, betibeglogene autotemcel should not be administered to women who are pregnant, and pregnancy after betibeglogene autotemcel infusion should be discussed with the treating physician. No nonclinical germline transmission studies have been conducted with betibeglogene autotemcel.
In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.
It is not known whether betibeglogene autotemcel has the potential to be transferred to the fetus. Therefore, betibeglogene autotemcel should not be administered to women who are pregnant, and pregnancy after betibeglogene autotemcel infusion should be discussed with the treating physician. No nonclinical germline transmission studies have been conducted with betibeglogene autotemcel.
In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively.
There is no information regarding the presence of betibeglogene autotemcel in human milk, the effect on the breastfed infant, and the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for betibeglogene autotemcel and any potential adverse effects on the breastfed child from betibeglogene autotemcel. Therefore, betibeglogene autotemcel is not recommended for women who are breastfeeding, and breastfeeding after betibeglogene autotemcel infusion should be discussed with the treating physician.
Betibeglogene autotemcel has not been studied in patients >65 years of age. Hematopoietic stem cell (HSC) transplantation must be appropriate for a patient to be treated with betibeglogene autotemcel.
The following prioritized warning is available for ZYNTEGLO (betibeglogene autotemcel):
No warning message for this drug.
No warning message for this drug.
The following icd codes are available for ZYNTEGLO (betibeglogene autotemcel)'s list of indications:
| Anemia in beta thalassemia | |
| D56.1 | Beta thalassemia |
| D56.2 | Delta-beta thalassemia |
| D56.3 | Thalassemia minor |
| D56.5 | Hemoglobin e-beta thalassemia |
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