Description
Marnetegragene autotemcel injection is used to treat severe leukocyte adhesion deficiency-I (LAD-I) caused by biallelic variants in ITGB2 in patients without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant (HSCT).
This medicine is to be given only by or under the direct supervision of your doctor.
This product is available in the following dosage forms:
- Suspension
Before Using
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.
Pediatric
Appropriate studies performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of marnetegragene autotemcel injection in children. Safety and efficacy have been established.
Geriatric
Appropriate studies on the relationship of age to the effects of marnetegragene autotemcel injection have not been performed in the geriatric population. Safety and efficacy have not been established.
Breastfeeding
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.
Drug Interactions
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. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
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.
- Infection (eg, HIV), active or untreated—Use is not recommended in patients with this condition.
- Liver disease—Use with caution. May make this condition worse.
Proper Use
A doctor or other trained health professional will give your child this medicine in a hospital. It is given through an IV catheter that is placed into a vein. This medicine must be given slowly, so the IV will need to stay in place for less than 10 minutes.
This medicine is made specifically for each patient, using the patient's own blood cells. Your child's doctor will collect your child's blood through a procedure called mobilization and apheresis. Talk with your child's doctor if you have any concerns about this.
Your child should not receive medicine to treat HIV for at least 1 month before the infusion.
Precautions
It is very important that your child's doctor check your child's progress at regular visits to make sure that this medicine is working properly. Blood tests are needed to check for unwanted effects.
The doctor may do tests to make sure your adolescent is not pregnant before this infusion.
Check with your child's doctor right away if your child has cough or hoarseness, fever or chills, lower back or side pain, or painful or difficult urination. These may be symptoms of an infection.
Check with your child's doctor right away if your child has pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, unusual tiredness or weakness, or yellow eyes or skin. These could be symptoms of a serious liver problem (eg, veno-occlusive disease).
This medicine lowers the number of some types of blood cells in your child's body. Because of this, your child may bleed or get infections more easily. To help with these problems, help your child avoid being near people who are sick or have infections. Have your child wash his or her hands often. Have your child stay away from rough sports or other situations where he or she could be bruised, cut, or injured. Brush and floss your child's teeth gently. Be careful when using sharp objects, including razors and fingernail clippers.
This medicine may increase your child's risk for cancer after treatment with this medicine. Talk to your doctor if you have concern about this.
Your child should not have any immunizations (vaccines) while being treated with this medicine without your doctor's approval. 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.
This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Check with your child's doctor right away if your child has chest tightness, cough, difficulty swallowing, fast heartbeat, hives, itching, skin rash, puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue, trouble breathing, or unusual tiredness or weakness.
Before your child has any medical tests (eg, HIV test), tell the medical doctor in charge that your child is taking this medicine. The results of some tests may be affected by this medicine.
Your child should not donate blood, organs, tissues, or cells at any time in the future.
Side Effects
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
- bladder pain
- bloody or cloudy urine
- body aches or pain
- chills
- cold or flu-like symptoms
- cough
- cough or hoarseness
- difficult, burning, or painful urination
- ear congestion
- fever
- frequent urge to urinate
- headache
- loss of voice
- lower back or side pain
- painful or difficult urination
- pale skin
- sneezing
- sore throat
- stuffy or runny nose
- trouble breathing
- ulcers, sores, or white spots in the mouth
- unusual bleeding or bruising
- unusual tiredness or weakness
Incidence not known
- Bloated stomach
- chest tightness
- difficulty swallowing
- dizziness
- fast heartbeat
- hives, itching, skin rash
- pain and fullness in right upper abdomen or stomach
- puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
- weight gain
- yellow eyes and skin
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
- Bumps on the skin
- constipation
- diarrhea
- loss of appetite
- loss or thinning of the hair
- nausea
- stomach pain
- 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.
DRG-80010476
