General MedicinePrescription OnlyPregnancy Cat. Not classifiedFDA Yes
Zynteglo
BETIBEGLOGENE AUTOTEMCEL
1 INDICATIONS AND USAGE ZYNTEGLO is indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions. ZYNTEGLO is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions. ( 1 )
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Brand names:Zynteglo
Quick Reference
Medicine ClassGeneral Medicine
FDA ApprovedYes
PregnancyCategory Not classified
Quick Facts
- Active ingredient
- BETIBEGLOGENE AUTOTEMCEL
- Medicine class
- General Medicine
- Brand names
- Zynteglo
- Availability
- Prescription only (Rx)
- Pregnancy category
- Not classified
- FDA approved
- Yes
What Is Zynteglo?
1 INDICATIONS AND USAGE ZYNTEGLO is indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions. ZYNTEGLO is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions. ( 1 )
Zynteglo (BETIBEGLOGENE AUTOTEMCEL) belongs to the General Medicine class of medications. It was first approved by the FDA in Yes. This medication requires a prescription from a licensed healthcare provider.
Key Safety Information
- •Always follow the dose recommended by your healthcare professional. Taking more than prescribed increases the risk of side effects including serious complications.
- •Seek emergency care if you experience signs of an allergic reaction — hives, swelling, difficulty breathing, or rash. Stop the medication and call your doctor immediately.
- •Some medications can increase the risk of cardiovascular events. Tell your doctor if you have high blood pressure, a history of heart attack or stroke, or any pre-existing health condition.
- •Avoid or limit alcohol while taking Zynteglo. Do not drink alcohol without first discussing it with your healthcare provider as it may increase adverse effects.
- •Take Zynteglo with food or a full glass of water if stomach upset occurs. Stop use and talk to your doctor if you experience persistent stomach pain or GI symptoms.
- •Tell your doctor about all medications, supplements, and vitamins you take. Some combinations significantly increase the risk of harm.
This is a summary only. Always read the full prescribing information and consult your healthcare provider for personalized medical advice.
Uses & Indications
Zynteglo is prescribed for the following conditions. Some uses are FDA-approved indications; others may be evidence-based off-label uses. Consult your healthcare provider for personalized guidance.
1 INDICATIONS AND USAGE ZYNTEGLO is indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions.
ZYNTEGLO is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with β-thalassemia who require regular red blood cell (RBC) transfusions.
Dosage Guide
The following are general dosing guidelines only. Your actual dose should be determined by your healthcare provider based on your condition, renal/hepatic function, and other medications.
AD
Adult Dosage
2 DOSAGE AND ADMINISTRATION For autologous use only. For one-time single-dose intravenous use only. For autologous use only. For intravenous use only. Patients are required to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for ZYNTEGLO manufacturing. ( 2.2 ) Dosing of ZYNTEGLO is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight. ( 2.1 ) The minimum recommended dose is 5.0 × 10 6 CD34+ cells/kg. ( 2.1 ) Full myeloablative conditioning must be administered before infusion of ZYNTEGLO. ( 2.2 ) Prophylaxis for hepatic veno-occlusive disease (VOD) is recommended. Prophylaxis for seizures should be considered. ( 2.2 ) Verify that the patient's identity matches the unique patient identification information on the ZYNTEGLO infusion bag(s) prior to infusion. ( 2.2 ) Do not sample, alter, or irradiate ZYNTEGLO. ( 2.2 ) Do not use an in-line blood filter or an infusion pump. ( 2.3 ) Administer each infusion bag of ZYNT
Available Forms
Available Strengths
Side Effects
Common
- 6 ADVERSE REACTIONS The following adverse reactions are described elsewhere in the labeling: Delayed Platelet Engraftment [see Warnings and Precautions (5.1) ] Risk of Neutrophil Engraftment Failure [see Warnings and Precautions (5.2) ] Risk of Insertional Oncogenesis [see Warnings and Precautions (5.3) ] Hypersensitivity Reactions [see Warnings and Precautions (5.4) ] 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).
- ( 6.1 ) The most common Grade 3 or 4 laboratory abnormalities (> 50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.
- ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genetix Biotherapeutics at 1-833-999-6378 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.
- The safety data described in this section reflect exposure to ZYNTEGLO in two open-label, single-arm clinical trials and one long-term follow-up study, in which 41 patients with β-thalassemia requiring regular transfusions were treated with ZYNTEGLO [see Clinical Studies (14) ].
- The median (min, max) age across the trials was 13 (4, 34) years;
- 49% were females;
Serious
- 5 WARNINGS AND PRECAUTIONS Delayed Platelet Engraftment: Monitor platelet counts until platelet engraftment and recovery are achieved.
- Patients should be monitored for thrombocytopenia and bleeding.
- ( 5.1 ) Risk of Neutrophil Engraftment Failure: Monitor absolute neutrophil counts (ANC) after ZYNTEGLO infusion.
- If neutrophil engraftment does not occur administer rescue cells.
- ( 5.2 ) Risk of Insertional Oncogenesis: Monitor patients at least annually for hematologic malignancies for at least 15 years after ZYNTEGLO infusion.
Rare
Medicine Interactions
Always inform your healthcare provider and pharmacist about ALL medications you take, including prescriptions, OTC medicines, vitamins, and supplements.
MajorModerateMinor
Warnings & Contraindications
5 WARNINGS AND PRECAUTIONS Delayed Platelet Engraftment: Monitor platelet counts until platelet engraftment and recovery are achieved. Patients should be monitored for thrombocytopenia and bleeding. ( 5.1 ) Risk of Neutrophil Engraftment Failure: Monitor absolute neutrophil counts (ANC) after ZYNTEGLO infusion. If neutrophil engraftment does not occur administer rescue cells. ( 5.2 ) Risk of Inser
How Zynteglo Works
12.1 Mechanism of Action ZYNTEGLO adds functional copies of a modified β-globin gene into patients' hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells with BB305 LVV. After ZYNTEGLO infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate to produce RBCs containing biologically active β A-T87Q -globin (a modified β-globin protein) that will combine with α-globin to produce functional adult Hb containing β A-T87Q -globin (HbA T87Q ). β A-T87Q -globin can be quantified relative to other globin species in peripheral blood using high-performance liquid chromatography. β A-T87Q -globin expression is designed to correct the β/α-globin imbalance in erythroid cells of patients with β-thalassemia and has the potential to increase functional adult HbA
Pharmacokinetics
Absorption
absorption, distribution, metabolism, and elimination are not applicable
Metabolism
metabolism, and elimination are not applicable
Pregnancy & Breastfeeding Safety
Not classified
Breastfeeding
Many medications pass into breast milk in varying amounts. Before using Zynteglowhile breastfeeding, discuss the benefits and risks with your healthcare provider or pharmacist — they can weigh your dose, your infant's age, and available lactation safety data to find the safest option for you and your baby.
Storage & Handling
Match the identity of the patient with the patient identifiers on the metal cassette(s), infusion bag(s), and Lot Information Sheet upon receipt. Keep the infusion bag(s) in the metal cassette(s) and store in the vapor phase of liquid nitrogen at less than or equal to -140°C (≤ -220°F) until ready f
Frequently Asked Questions
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Adult, pediatric, renal, and hepatic dosing for Zynteglo
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Zynteglo side effects
Complete adverse effect profile including common, serious, and rare reactions
Sources & References
- • FDA Prescribing Information — Official labeling and package insert for Zynteglo
- • U.S. National Library of Medicine — MedlinePlus drug information
- • American Hospital Formulary Service (AHFS) — Clinical pharmacology monograph
- • PubMed / MEDLINE — View clinical studies
Last reviewed by MedCentralHub Medical Review Board · MedCentralHub Editorial Policy
Medical Disclaimer
The information on this page is for educational purposes only and is not intended as medical advice, diagnosis, or treatment. Always consult your doctor, pharmacist, or qualified healthcare provider before starting, stopping, or changing any medication.