Description
Atidarsagene autotemcel injection is used to treat children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
Metachromatic leukodystrophy (MLD) is a rare genetic disorder that affects the brain and nervous system caused by a deficiency of an enzyme called arylsulfatase A (ARSA).
This medicine is to be given only by or under the immediate 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 atidarsagene autotemcel injection in children. However, safety and efficacy have not been established in children with late juvenile MLD.
Geriatric
No information is available on the relationship of age to the effects of atidarsagene autotemcel injection in geriatric patients.
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. 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.
- Abacavir
- Abacavir Sulfate
- Adenovirus Vaccine Type 4, Live
- Adenovirus Vaccine Type 7, Live
- Amprenavir
- Anthrax Vaccine Adsorbed
- Atazanavir
- Atazanavir Sulfate
- Bacillus of Calmette and Guerin Vaccine, Live
- Cabotegravir
- Cholera Vaccine
- Cholera Vaccine, Live
- Cobicistat
- Darunavir
- Darunavir Ethanolate
- Delavirdine
- Delavirdine Mesylate
- Dengue Tetravalent Vaccine, Live
- Didanosine
- Diphtheria Toxoid, Adsorbed
- Dolutegravir
- Dolutegravir Sodium
- Doravirine
- Ebola Zaire Vaccine, Live
- Efavirenz
- Elvitegravir
- Emtricitabine
- Enfuvirtide
- Etravirine
- Fosamprenavir
- Fosamprenavir Calcium
- Fostemsavir
- Haemophilus B Vaccine
- Hepatitis A Vaccine, Inactivated
- Hepatitis B Vaccine
- Human Papillomavirus Vaccine
- Ibalizumab-uiyk
- Indinavir
- Indinavir Sulfate
- Influenza Virus Vaccine
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.
- Kidney disease or
- Liver disease—Use with caution. This medicine has not been studied in patients with these conditions.
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 in a vein. This medicine must be given slowly, so the IV will need to stay in place for at least 30 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 use medicine to treat HIV for at least 1 month before having a procedure to collect his or her blood.
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 having a procedure to collect blood. Female patients and male patients with female partners should use an effective form of birth control before having a procedure to collect blood and for at least 6 months after the last dose.
This medicine may increase your child's risk for blood clots. Check with your child's doctor right away if your child has pain in the chest, groin, or legs, especially the calves, severe headache, slurred speech, sudden, unexplained trouble breathing, sudden loss of coordination, sudden, severe weakness or numbness in the arm or leg, tenderness, pain, swelling, warmth, skin discoloration, and prominent superficial veins over affected area, or vision changes.
Tell your child's doctor right away if your child has a headache, confusion, seizures, stiff neck, or vomiting while receiving this medicine. These may be symptoms of encephalitis.
This medicine may cause serious infections. Check with your child's doctor right away if your child has fever or chills, cough or hoarseness, lower back or side pain, or painful or difficult urination.
Atidarsagene autotemcel will lower 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, avoid being near people who are sick or have infections. Wash your and your child's hands often. Stay away from rough sports or other situations where your child could be bruised, cut, or injured. Tell your child to brush and floss teeth gently. Your child should be careful when using sharp objects, including razors and fingernail clippers. Call your child's doctor right away if your child has fever, chills, or any signs or 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, vomiting, or yellow eyes or skin. These could be symptoms of a serious liver problem (eg, hepatic veno-occlusive disease).
This medicine may cause serious allergic reaction, including anaphylaxis, which may be life-threatening and requires immediate medical attention. Check with your child's doctor right away if your child has a rash, itching, dizziness, fainting, fast heartbeat, trouble breathing or swallowing, or chest tightness while using this medicine.
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.
Your child should not donate blood, organs, tissues, or cells at any time in the future.
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.
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
- body aches or pain
- chest pain or tightness
- chills
- confusion
- cough
- dizziness
- ear congestion
- fainting
- fast heartbeat
- fever
- headache
- lightheadedness
- 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
- difficulty in swallowing
- pain and fullness in the right upper abdomen or stomach
- pain in the chest, groin, or legs, especially the calves
- skin itching, rash, or redness
- slurred speech
- sudden, severe weakness or numbness in the arm or leg
- sudden loss of coordination
- swelling of the face, throat, or tongue
- tenderness, pain, swelling, warmth, skin discoloration, and prominent superficial veins over affected area
- vision changes
- 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
- Blistering, crusting, irritation, itching, or reddening of the skin
- cracked, dry, scaly skin
- diarrhea
- loss of appetite
- nausea
- stomach pain
- swelling or inflammation of the mouth
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.
CON-XXXXXXXX
