Pronunciation:

el-i-VAL-doe-jeen aw-toe-TEM-sel

Brand Names:

• Skysona

Dosage Forms:

• Suspension

Warnings:

Hematologic malignancy, including life-threatening cases of myelodysplastic syndrome, has occurred in patients treated with elivaldogene autotemcel. Patients have been diagnosed between 14 months and 7.5 years after elivaldogene autotemcel administration, and the cancers appear to be the result of the elivaldogene autotemcel lentiviral vector, Lenti-D, integration in proto-oncogenes. Monitor patients closely for evidence of malignancy through complete blood counts at least every 6 months and through assessments for evidence for clonal expansion or predominance at least twice in the first year and annually thereafter; consider bone marrow evaluations as clinically indicated .

Classifications:

Therapeutic—

Immunological Agent

Uses of This Medicine:

Elivaldogene autotemcel injection is used to treat early, active cerebral adrenoleukodystrophy (CALD) in boys 4 to 17 years of age.

Cerebral adrenoleukodystrophy (CALD) is a genetic disease caused by mutations in the ABCD1 gene that lead to the buildup of very long fatty acids (VLCFAs) in the brain. These fatty acids may cause brain and nerve damage. The damage can be seen on magnetic resonance imaging (MRI) scans of the brain. Elivaldogene autotemcel injection is recommended if the damage is determined to be early and if the brain disease is active. It will help the body to break down the fatty acids to slow the progression of damage to the brain and slow the decline in nerve function.

This medicine is to be given only by or under the immediate supervision of your doctor.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of elivaldogene autotemcel injection in children younger than 4 years of age. Safety and efficacy have not been established.

Older adults—

No information is available on the relationship of age to the effects of elivaldogene autotemcel injection in geriatric patients.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are receiving this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Adenovirus Vaccine, Live
• Anthrax Vaccine Adsorbed
• Bacillus of Calmette and Guerin Vaccine, Live
• Cholera Vaccine
• Cholera Vaccine, Live
• Dengue Tetravalent Vaccine, Live
• Diphtheria Toxoid, Adsorbed
• Ebola Zaire Vaccine, Live
• Haemophilus B Vaccine
• Hepatitis A Vaccine, Inactivated
• Hepatitis B Vaccine
• Human Papillomavirus Vaccine
• Influenza Virus Vaccine
• Influenza Virus Vaccine, Live
• Japanese Encephalitis Virus Vaccine
• Measles Virus Vaccine, Live
• Meningococcal Polysaccharide Vaccine
• Meningococcal Vaccine
• Mumps Virus Vaccine, Live
• Pertussis Vaccine
• Pneumococcal Vaccine
• Pneumococcal Vaccine, Diphtheria Conjugate
• Pneumococcal Vaccine Polyvalent
• Poliovirus Vaccine, Inactivated
• Poliovirus Vaccine, Live
• Rabies Vaccine
• Rotavirus Vaccine, Live
• Rubella Virus Vaccine, Live
• SARS-COV-2 (COVID-19) Vaccine, Adenovirus 26 Vector (Janssen)
• SARS-COV-2 (COVID-19) Vaccine, mRNA (Moderna)
• SARS-COV-2 (COVID-19) Vaccine, mRNA (Pfizer)
• SARS-COV-2 (COVID-19) Vaccine, Protein Subunit, Adjuvanted (Novavax)
• SARS-COV-2 (COVID-19) Vaccine, Protein Subunit, Adjuvanted (Sanofi)
• Smallpox Monkeypox Vaccine, Live Non-Replicating
• Smallpox Vaccine
• Tetanus Toxoid
• Tick-Borne Encephalitis Vaccine
• Typhoid Vaccine
• Typhoid Vaccine, Live
• Typhoid Vi Polysaccharide Vaccine
• Varicella Virus Vaccine
• Varicella Virus Vaccine, Live
• Yellow Fever Vaccine
• Zoster Vaccine, Live

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Allergy to dimethyl sulfoxide (DMSO), history of—Use with caution. May increase the risk of an allergic reaction.

• Infections, active—Should not be used in patients with this condition.

• Kidney disease or
• Liver disease—Use with caution. This medicine has not been studied in patients with these conditions.

Proper Use of This Medicine:

A doctor or other trained health professional will give you this medicine in a hospital. It is given through a needle placed in a vein. This medicine must be given slowly, so your IV tube will need to stay in place for at least 60 minutes.

This medicine comes with a Medication Guide. Read and follow these instructions carefully. Ask your doctor or pharmacist if you have any questions.

You may also receive other medicines (eg, cancer medicine, fever medicine, allergy medicine, or medicine to prevent nausea or vomiting) before receiving this medicine.

This medicine is made specifically for each patient, using the patient's own blood cells. Your doctor will collect your blood through a procedure called mobilization and apheresis. Talk with your doctor if you have any concerns about this.

Your doctor will also want you to stay in the hospital or center for at least 2 months after your infusion.

Do not use medicine to treat HIV for at least 1 month before having a procedure to collect your blood.

Precautions While Using This Medicine:

It is very important that your doctor check your progress at regular visits to make sure that this medicine is working properly. Blood tests are needed every 6 months for at least 15 years to check for unwanted effects.

Male patients with female partners should use an effective form of birth control before having a procedure to collect your blood and for at least 6 months after the last dose.

This medicine may cause blood cancer (eg, myelodysplastic syndrome). Check with your doctor right away if you have chest pain, chills, cough or hoarseness, fever, lower back or side pain, painful or difficult urination, sores, ulcers, or white spots on the lips or in the mouth, swollen glands, unusual bleeding or bruising, or unusual tiredness or weakness.

Elivaldogene autotemcel will lower the number of some types of blood cells in your body. Because of this, you may bleed or get infections more easily. To help with these problems, avoid being near people who are sick or have infections. Wash your hands often. Stay away from rough sports or other situations where you could be bruised, cut, or injured. Brush and floss your teeth gently. Be careful when using sharp objects, including razors and fingernail clippers. Call your doctor right away if you have fever, chills, or any signs or symptoms of an infection.

This medicine may cause serious allergic reaction, including anaphylaxis, which may be life-threatening and requires immediate medical attention. Check with your doctor right away if you have a rash, itching, dizziness, fainting, fast heartbeat, trouble breathing or swallowing, or chest tightness while you are using this medicine.

While you are being treated with elivaldogene autotemcel, do not have any immunizations (vaccinations) without your doctor's approval. Live virus vaccinations should not be given for at least 6 weeks before start of treatment, during treatment, and until recovery following the last cycle of this medicine.

Do not donate blood, organs, tissues, or cells during treatment with this medicine.

Before you have any medical tests (eg, HIV test), tell the medical doctor in charge that you are taking this medicine. The results of some tests may be affected by this medicine.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

Black, tarry stools

bleeding gums

blood in the urine or stools

blurred vision

chest pain

chills

cough or hoarseness

dizziness

fast, pounding, or irregular heartbeat or pulse

fever

headache

lower back or side pain

nervousness

painful or difficult urination

pale skin

pinpoint red spots on the skin

pounding in the ears

seizures

slow or fast heartbeat

sore throat

sores, ulcers, or white spots on the lips or in the mouth

swollen glands

trouble breathing

unusual bleeding or bruising

unusual tiredness or weakness

Incidence not known

Chest tightness

difficulty swallowing

hives, itching, or skin rash

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Back pain

bloody nose

constipation

cracked lips

darkening of the skin

decreased appetite

diarrhea

fear

flushing

irritation or soreness of the mouth

loss or thinning of the hair

nausea

stomach pain

swelling or inflammation of the mouth

vomiting

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.