Elivaldogene autotemcel Injection
Elivaldogene autotemcel (Skysona) is a one-time gene therapy given intravenously to slow the progression of neurologic damage in boys aged 4–17 with early, active cerebral adrenoleukodystrophy (CALD) who do not have a matched bone marrow donor available.
🧬 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 →
⚠️ FDA Boxed Warning — the FDA’s strongest safety alert ▾
Skysona carries a serious boxed warning: it can cause life-threatening blood cancers, including myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). These cancers have developed in patients treated with Skysona in clinical studies, appearing as early as 14 months and as late as 10 years after the infusion. The cancers appear to be directly caused by how the therapy inserts its corrective gene into the patient's DNA. All patients who receive Skysona must be monitored for the rest of their lives with regular blood counts (at least every 3 months) and specialized tests to detect abnormal cell growth at least twice in the first year, then every year after that. Watch for signs such as persistent fatigue, unusual bruising or bleeding, frequent infections, or unexplained weight loss, and report them to your doctor right away.
📖 Elivaldogene autotemcel Injection: Patient Information Guide
A plain-language walkthrough of Elivaldogene 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▾
Skysona (elivaldogene autotemcel) is the only product in this class; all supported uses below apply to Skysona given as an intravenous infusion.
- Skysona is indicated to slow the progression of neurologic dysfunction in boys ages 4–17 with early, active cerebral adrenoleukodystrophy (CALD) who do not have a matched HLA donor available for a standard allogeneic (donor) stem cell transplant.
- Early, active CALD means the boy is asymptomatic or mildly symptomatic (neurologic function score ≤ 1), has gadolinium enhancement on brain MRI, and has a Loes score (a measure of brain damage) of 0.5–9.
- This approval is accelerated — based on 24-month disability-free survival data — and confirmation of long-term clinical benefit is still being studied.
- Skysona does not treat or prevent adrenal insufficiency, a separate complication of adrenoleukodystrophy that requires its own management.
⚙️How it works▾
In CALD, a faulty ABCD1 gene means the body cannot make a working version of a protein called ALDP, which normally breaks down very long chain fatty acids (VLCFAs). Without ALDP, VLCFAs build up and trigger inflammation that destroys the myelin sheath — the protective coating around nerves in the brain. Skysona works by taking a patient's own blood stem cells, inserting a functional copy of the ABCD1 gene into them in the laboratory using a viral carrier (the Lenti-D lentiviral vector), and then infusing those corrected cells back into the patient. Once they settle in the bone marrow, the corrected cells produce working ALDP, which can then participate in clearing the damaging fatty acids and is believed to slow or possibly prevent further brain inflammation and demyelination.
💊How it's taken▾
Skysona is given as a single, one-time intravenous infusion — there is no repeated dosing schedule. The amount infused is based on the number of corrected stem cells collected per kilogram of the patient's body weight; the minimum recommended amount is 5.0 × 10⁶ CD34+ cells per kg. Before the infusion, the patient's own stem cells must be collected through a process called apheresis, genetically modified in a specialized laboratory, and returned to the treatment center. Full myeloablative (bone-marrow-clearing) conditioning chemotherapy is given first, followed by at least 48 hours of washout before Skysona is infused. All preparation, infusion, and post-treatment steps are managed entirely by the specialized care team.
🤒Side effects — in detail▾
Because Skysona involves intensive conditioning chemotherapy as well as the gene therapy infusion itself, many side effects occur in the weeks surrounding treatment. Common reactions include:
- Mouth sores (mucositis) — very common, affecting most patients
- Nausea, vomiting, and decreased appetite
- Febrile neutropenia (fever with dangerously low white blood cell counts)
- Hair loss (alopecia)
- Abdominal pain and constipation
- Headache and skin rash
- Low blood counts (leukopenia, thrombocytopenia, anemia, neutropenia) — nearly universal and may persist for more than a year
- Low potassium levels (hypokalemia)
Serious reactions to act on immediately include signs of blood cancer (persistent fatigue, unexplained bruising or bleeding, frequent infections), signs of a severe allergic reaction (difficulty breathing, hives, sudden blood pressure drop), and any new or worsening neurologic symptoms such as seizures or vision changes.
⚠️Warnings & precautions▾
- Blood cancer (hematologic malignancy): This is the most critical risk. Skysona's gene-delivery mechanism can insert near cancer-promoting genes in the DNA, and 15% of clinical study patients developed MDS or AML — some as late as 10 years after treatment. Lifelong blood monitoring is required.
- Serious and fatal infections: The conditioning chemotherapy and period of low immunity after infusion create a window for severe bacterial, viral, and fungal infections. Febrile neutropenia is common and requires immediate medical evaluation.
- Prolonged low blood counts (cytopenias): Low platelet, white blood cell, or red blood cell counts can persist for more than a year, increasing bleeding and infection risk.
- Delayed platelet engraftment: Some patients take much longer than expected to rebuild their platelet counts, raising bleeding risk; close monitoring is needed until counts recover.
- Neutrophil engraftment failure: In rare cases the immune-fighting white cells (neutrophils) may not recover adequately; rescue stem cells collected before treatment may be needed.
- Hypersensitivity reactions: The preservative DMSO in Skysona can cause allergic reactions, including potentially life-threatening anaphylaxis, during the infusion.
- False-positive HIV test: Skysona interferes with standard PCR-based HIV tests; different testing methods must be used after treatment.
🔀What it interacts with▾
Skysona has a small but important list of interactions to be aware of before and after treatment:
- Anti-retroviral medications (HIV drugs): Must be stopped at least one month before starting stem cell mobilization and kept stopped until all apheresis cycles are complete — they can prevent the gene therapy from being manufactured correctly.
- Vaccines: Live and non-live vaccines should be avoided for at least 6 weeks before conditioning chemotherapy and until blood counts have fully recovered after Skysona, as the immune system cannot respond normally during this period. Childhood vaccines should ideally be completed before conditioning begins.
- PCR-based HIV tests: Skysona's lentiviral vector causes a false-positive result on standard PCR HIV tests — this is not a true infection. Alternative HIV testing methods must be used going forward.
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▾
- Age under 4: Safety and effectiveness of Skysona have not been established in boys younger than 4 years.
- Full ABCD1 gene deletion: Patients with a complete deletion of this gene may lose Skysona's benefit rapidly due to an immune response against the modified cells.
- Active infection: Skysona should not be given to a patient with an active infection; treat the infection first.
- HIV-positive patients: Skysona cannot be manufactured from the cells of a patient with HIV; a negative HIV test is required before cell collection.
- Kidney or liver problems: Skysona has not been studied in patients with renal or hepatic impairment; suitability for stem cell transplantation must be assessed individually.
- Fertility: The conditioning chemotherapy carries a risk of permanent infertility; discuss sperm banking before treatment begins if appropriate.
- Pregnancy and breastfeeding: No safety data exist. Effective contraception (IUD or combined hormonal plus barrier method) is required from the start of mobilization through at least 6 months after Skysona infusion.
- Blood donation: Patients who have received Skysona must never donate blood, organs, tissues, or cells.
📡Pharmacodynamics — effects in the body▾
After Skysona is infused, the corrected stem cells begin producing ALDP — the protein missing in CALD — in blood cells called monocytes (CD14+ cells). This production is detectable within the first month and typically stabilizes by about 6 months after infusion. Studies show ALDP-producing cells remain present at meaningful levels through at least 24 months, and some patients show ongoing ALDP expression at 5 years, indicating the corrected cells can persist long-term in the body.
🔬Pharmacokinetics — how your body processes it▾
Skysona is an autologous (patient-derived) gene therapy made of living, genetically modified stem cells. Because it is not a small-molecule drug, conventional measurements of absorption, distribution, metabolism, and elimination do not apply — the therapy engrafts in the bone marrow and becomes part of the patient's own cell population rather than being processed and cleared like a traditional medicine.
📦How to store it▾
Store SKYSONA frozen 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 Elivaldogene 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 Elivaldogene autotemcel Injection — the kind of thing our pharmacy team would talk through with you at the counter.
What exactly is Skysona and how is it different from regular medicine? ▾
Why does the label say there's a cancer risk? Should we be scared? ▾
What should we expect in the weeks right after the infusion? ▾
Does Skysona cure everything related to adrenoleukodystrophy? ▾
Are there any medications or vaccines my son needs to avoid before or after Skysona? ▾
What signs should we watch for after treatment that mean we need to call the doctor right away? ▾
When was Elivaldogene autotemcel Injection first available? ▾
Who makes Elivaldogene autotemcel Injection? ▾
💬 Answers drafted from Elivaldogene 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 Elivaldogene autotemcel Injection comes
Elivaldogene 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
- Skysona is used to slow neurologic deterioration in boys ages 4–17 with early, active cerebral adrenoleukodystrophy (CALD) who do not have a matched stem cell donor
- Skysona is approved under accelerated approval based on 24-month data; continued approval depends on confirmatory trial results
- Skysona is given as a single intravenous infusion by gravity drip over less than 60 minutes at a specialized treatment center, after the patient has undergone stem cell collection and conditioning chemotherapy
- Skysona must be thawed at 37°C and infused within 4 hours of thawing; do not use an in-line blood filter or infusion pump
- Before infusing Skysona, the patient's identity must be confirmed against the unique identifiers on every infusion bag
- After the full contents of each Skysona bag are infused, flush the bag and tubing with at least 50 mL of normal saline to deliver as many cells as possible
📊 Elivaldogene autotemcel Injection across the U.S. market
How Elivaldogene 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 Elivaldogene autotemcel Injection products
A representative set of FDA-listed product records for Elivaldogene 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 | ||||
| 30000000 1 | Intravenous | bluebird bio, Inc. | BLA | View NDC → |
| 30000000 1-Jan | Intravenous | bluebird bio, Inc. | BLA | View NDC → |
| ELIVALDOGENE AUTOTEMCEL 30000000 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 Elivaldogene 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 Elivaldogene 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 Elivaldogene 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 Elivaldogene 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 2612527 1 dose form · 1 strength
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Dose form (SCDF) Injection RxCUI 2612551In current FDA listingsClinical drug / strength (SCD) 20 ML elivaldogene autotemcel 30000000 CELLS/MLRxCUI 2612552
Look up RxCUI 2612527 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 Elivaldogene 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
Elivaldogene autotemcel Injection belongs to the Various alimentary tract and metabolism products 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.