Betibeglogene autotemcel Injection
Betibeglogene autotemcel (Zynteglo) is a one-time gene therapy given by intravenous infusion to treat β-thalassemia in adults and children who need regular red blood cell transfusions.
🧬 Where this information comes from Click to expand
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 1 clinical label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
📖 Betibeglogene autotemcel Injection: Patient Information Guide
A plain-language walkthrough of Betibeglogene autotemcel Injection — what it treats, how it works, how it’s taken, side effects, warnings and interactions. Tap any section to expand it.
🎯What it's used for▾
Betibeglogene autotemcel (Zynteglo) is a gene therapy approved for one specific blood disorder:
- β-thalassemia requiring regular red blood cell transfusions — Zynteglo is indicated for adult and pediatric patients (age 4 and older) with this genetic hemoglobin disorder who depend on regular transfusions. It is a one-time treatment, delivered as a single IV infusion at a qualified treatment center.
⚙️How it works▾
β-thalassemia is caused by a faulty or missing β-globin gene, which means the body cannot make enough working hemoglobin to carry oxygen in red blood cells. Zynteglo works by adding a corrected, functional copy of a modified β-globin gene into the patient's own blood stem cells (called CD34+ hematopoietic stem cells) outside the body. Once those corrected cells are infused back, they travel to the bone marrow, take up permanent residence, and begin producing red blood cells that contain a working form of hemoglobin called HbA T87Q. This corrects the imbalance between α-globin and β-globin that drives the disease, with the potential to raise hemoglobin levels to near-normal and end the need for regular transfusions.
💊How it's taken▾
Zynteglo is not a daily medication — it is a one-time, single-dose intravenous infusion. The dose is based on the number of corrected stem cells in the infusion bag(s), calculated per kilogram of body weight. Before the infusion, patients undergo stem cell mobilization and collection (apheresis), followed by strong chemotherapy conditioning to prepare the bone marrow. After conditioning, there is a mandatory wait of at least 48 hours before Zynteglo is infused. Each infusion bag is given over less than 30 minutes by IV; if more than one bag is provided, each is given one at a time. The entire process — from stem cell collection to infusion — takes place over several weeks at a specialized center.
- Do not miss follow-up appointments — monitoring continues for months and years after infusion.
- Do not donate blood, organs, tissues, or cells at any time after receiving Zynteglo.
🤒Side effects — in detail▾
The most common side effects seen in clinical trials (affecting at least 20% of patients) included:
- Mouth sores (mucositis) — very common and often severe
- Febrile neutropenia (fever with dangerously low infection-fighting white blood cells)
- Nausea, vomiting, and diarrhea
- Fever (pyrexia)
- Hair loss (alopecia)
- Nosebleeds (epistaxis)
- Abdominal pain and musculoskeletal pain
- Headache, rash, itching, decreased appetite, and skin color changes
Serious reactions requiring prompt attention include severe liver disease (veno-occlusive disease), sepsis, low platelet counts with bleeding, engraftment failure, and potential long-term risk of blood cancer. Tell your care team right away about any unusual bleeding, jaundice, high fever, or signs of a blood cancer such as swollen lymph nodes or unexplained bruising.
⚠️Warnings & precautions▾
- Delayed platelet engraftment: Platelets (clotting cells) can take weeks to recover after infusion, raising your bleeding risk. About 15% of patients had severe low platelets past Day 100. Frequent blood counts and close monitoring are essential.
- Neutrophil engraftment failure: In rare cases, the immune-rebuilding white blood cells (neutrophils) may not recover adequately. If this happens, rescue treatment using a back-up stem cell collection is required.
- Lifelong risk of blood cancer (insertional oncogenesis): Because the gene is permanently inserted into DNA, there is a small but real risk it could trigger a blood cancer at any point in life. Annual blood count monitoring is required for at least 15 years.
- Hypersensitivity / allergic reactions: The preservative DMSO in Zynteglo can cause allergic reactions, including serious anaphylaxis, during infusion. Monitor closely during and after each bag.
- False-positive HIV test: After receiving Zynteglo, PCR-based HIV tests will give a false positive result for life. Alert all future healthcare providers to this.
🔀What it interacts with▾
Zynteglo interacts with certain medications in ways that can affect both the manufacturing process and recovery after infusion:
- Anti-retroviral HIV medications: Must be stopped at least one month before stem cell mobilization and held until all cell collection is complete — they interfere with the genetic modification process.
- Hydroxyurea: Must also be stopped at least one month before mobilization and held through apheresis.
- Iron chelators (iron-removal drugs): Must be stopped at least 7 days before conditioning chemotherapy. After infusion, myelosuppressive (bone-marrow-suppressing) iron chelators should be avoided for 6 months. Non-myelosuppressive alternatives or phlebotomy (blood removal) may be used if iron removal is still needed.
- Live vaccines: Safety of live viral vaccines during or after Zynteglo treatment is unknown — follow your treatment center's guidance.
- Erythropoiesis-stimulating agents (ESAs): No data exist on using these red-blood-cell-boosting drugs alongside Zynteglo.
This is not a complete list of interactions. Always talk with your doctor or pharmacist before starting, stopping, or changing any medication.
📋Before taking it▾
- Pregnancy: Zynteglo should not be given to pregnant women. The conditioning chemotherapy also carries serious risks to pregnancy and fertility. A negative pregnancy test is required before mobilization, before conditioning, and before infusion.
- Breastfeeding: Zynteglo is not recommended during breastfeeding; discuss with your doctor.
- Fertility: The conditioning chemotherapy can cause infertility. Talk to your doctor about banking sperm or eggs before starting treatment.
- Contraception: Use effective birth control from the start of mobilization through at least 6 months after infusion.
- Children under 4: Safety and effectiveness have not been established in this age group.
- Adults over 65: Zynteglo has not been studied in patients older than 65; suitability for stem cell transplantation must be assessed.
- HIV-positive patients: Zynteglo cannot be manufactured from the cells of someone who tests positive for HIV — a negative HIV test is required for cell collection to proceed.
- Kidney or liver problems: You will be evaluated to make sure a stem cell transplant is appropriate and safe for you.
📡Pharmacodynamics — effects in the body▾
After Zynteglo infusion, levels of the therapeutic hemoglobin (called HbA T87Q) rise gradually and generally stabilize around Month 6. In Phase 3 studies, the median HbA T87Q level at Month 6 was 8.7 g/dL, and at Month 24 it remained durable at a median of 8.8 g/dL. This effect appears to be long-lasting — data through Month 36 showed continued sustained expression, consistent with the gene being permanently integrated into long-lived stem cells.
🔬Pharmacokinetics — how your body processes it▾
Because Zynteglo is made from a patient's own genetically modified stem cells, standard pharmacokinetic concepts like absorption, distribution, metabolism, and elimination do not apply. The corrected stem cells engraft permanently in the bone marrow and produce the therapeutic protein indefinitely — this is not a drug that is processed and cleared from the body in the conventional sense.
📦How to store it▾
store in the vapor phase of liquid nitrogen at less than or equal to -140°C (≤ -220°F) until ready for thaw and administration.
📄 Written from Betibeglogene autotemcel Injection’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Every point is grounded in that labeling — nothing is invented. How we use AI →
🩺 Pharmacist Counseling Corner
Quick, plain-English answers to the questions patients ask most about Betibeglogene autotemcel Injection — the kind of thing our pharmacy team would talk through with you at the counter.
Will I only need this treatment once? ▾
What happens to my body before I actually get the infusion? ▾
What side effects should I expect after the infusion? ▾
I've heard this therapy could cause cancer — is that true? ▾
Can I take my other medications normally during this process? ▾
Will this affect future blood tests or medical procedures? ▾
When was Betibeglogene autotemcel Injection first available? ▾
Who makes Betibeglogene autotemcel Injection? ▾
💬 Answers drafted from Betibeglogene autotemcel Injection’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Grounded in that labeling — nothing is made up. How we use AI →
🧰 Keep exploring
💊 How Betibeglogene autotemcel Injection comes
Betibeglogene autotemcel Injection is available in 1 form. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Injection
How this form is used
- Zynteglo is used for the one-time treatment of β-thalassemia in adults and children who need regular red blood cell transfusions
- Zynteglo aims to reduce or eliminate dependence on regular transfusions by correcting hemoglobin production
- Zynteglo is given as a single intravenous infusion at a specialized treatment center — each infusion bag is administered over less than 30 minutes
- Zynteglo must be infused within 4 hours of thawing; do not use an in-line blood filter or infusion pump
- Zynteglo is for autologous use only — the patient's identity must be confirmed against the infusion bag label before administration
- Zynteglo requires myeloablative conditioning (strong chemotherapy) to be completed at least 48 hours before infusion
📊 Betibeglogene autotemcel Injection across the U.S. market
How Betibeglogene autotemcel Injection appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
Share of FDA-listed product records by manufacturer — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
🔍 Compare Betibeglogene autotemcel Injection products
A representative set of FDA-listed product records for Betibeglogene autotemcel Injection — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Injection | ||||
| 20000000 1 | Intravenous | bluebird bio, Inc. | BLA | View NDC → |
| 20000000 1-Jan | Intravenous | bluebird bio, Inc. | BLA | View NDC → |
| BETIBEGLOGENE AUTOTEMCEL 20000000 1 | Intravenous | Genetix Biotherapeutics Inc. × 2 listings | BLA | View NDC → |
Showing 4 of 4 products — FDA National Drug Code Directory. See every product, package size and price: browse all 4 Betibeglogene autotemcel Injection NDCs →
📈 What people report — real-world data (FDA FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Betibeglogene autotemcel Injection — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Betibeglogene autotemcel Injection in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
🏛️ The road to your pharmacy
How Betibeglogene autotemcel Injection went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
🚨 Recall history
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
Source: openFDA drug enforcement (recall) reports.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one ingredient concept (IN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). Combination products remain separate concepts with their own pages.
RxCUI 2610349 1 dose form · 1 strength
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Dose form (SCDF) Injection RxCUI 2610352In current FDA listingsClinical drug / strength (SCD) 20 ML betibeglogene autotemcel 20000000 CELLS/MLRxCUI 2610435
Look up RxCUI 2610349 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
📦 Supply & shortage status
Whether Betibeglogene autotemcel Injection is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
🏷️ Sold under these brand names
🏭 Manufacturers & labelers
RxNorm drug class
Betibeglogene autotemcel Injection belongs to the Other hematological agents class.
Classified by RxNorm RxClass — the U.S. National Library of Medicine's standardized drug-classification service (Established Pharmacologic Class from the FDA, the ATC drug family from the WHO, and mechanism of action). Reference only, not medical advice.
🔬 Where this comes from
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How we combine label and product data
HelloPharmacist combines public FDA and NLM records; we don’t author the underlying clinical facts. We identified 1 FDA-filed SPL label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. The representative label is the starting point for the displayed reference monograph; the 12 largest stored in-scope SPL records, which may include that representative, are compared section by section, and the longest available version of each section is retained. Forms, routes, brands, labelers, product-listed strengths, and product-record counts are aggregated separately from ingredient-matched FDA NDC Directory records; package records listed above are associated with the linked SPL; and the normalized concept hierarchy comes from NLM RxNorm.
Where a section is identified as AI-written, its plain-language text is generated from bounded label evidence. New draft drug pages remain in the admin review queue before first publication. bluebird bio, Inc. is the representative DailyMed label linked for verification and dates; it is not the sole source used for the page.
This is general education, not medical advice — always consult your doctor or pharmacist about your medications and any medical condition. For the exact wording of the linked representative label, view that label on DailyMed; some displayed composite sections may come from other in-scope SPL labels. Learn how we use AI and our data sources & update cadence.