Atidarsagene Autotemcel (at-i-DAR-sa-jeen aw-toe-TEM-sel)

Treats metachromatic leukodystrophy (MLD).

Brand Name(s):

Lenmeldy

When This Medicine Should Not Be Used:

How to Use This Medicine:

Injectable

• Your doctor will prescribe your dose and schedule. This medicine is given through a needle placed in a vein. This medicine must be given slowly, so the IV will need to stay in place for at least 30 minutes.
• A nurse or other health provider will give you this medicine.
• 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.

Drugs and Foods to Avoid:

Ask your doctor or pharmacist before using any other medicine, including over-the-counter medicines, vitamins, and herbal products.

• Your child should not use medicine to treat HIV for at least 1 month before having a procedure to collect his or her blood.
• This medicine may interfere with vaccines. Ask your doctor before you get a flu shot or any other vaccines. Your child should not receive vaccines at least 6 weeks before the start of treatment, during treatment, and until recovery following the last cycle of this medicine.

Warnings While Using This Medicine:

• If your adolescent can get pregnant, the doctor may do tests to make sure she 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.
• Tell the doctor if your adolescent is breastfeeding, or if your child has kidney disease, liver disease, or a history of dimethyl sulfoxide (DMSO) allergy.
• This medicine may cause the following problems:
  • Increased risk of blood clots
  • Increased risk of encephalitis (swelling of the brain)
  • Serious infections
  • Liver problems
  • Increased risk of blood cancer
• This medicine may make you bleed, bruise, or get infections more easily. Take precautions to prevent illness and injury. Wash your hands often.
• Your child should not donate blood, organs, tissues, or cells at any time in the future.
• Tell any doctor or dentist who treats you that you are using this medicine. This medicine may affect certain medical test results.
• Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments.

Possible Side Effects While Using This Medicine:

Call your doctor right away if you notice any of these side effects:

• Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
• Chest pain, trouble breathing, coughing up blood
• Confusion, irritability, headache, seizures, stiff neck
• Dark urine or pale stools, nausea, vomiting, loss of appetite, stomach pain, yellow skin or eyes
• Fever, chills, cough, sore throat, body aches
• Numbness or weakness in your arm or leg, or on one side of your body, pain in your lower leg
• Sores or white patches on your lips, mouth, or throat
• Sudden or severe headache, problems with vision, speech, or walking

If you notice these less serious side effects, talk with your doctor:

• Diarrhea, loss of appetite
• Pain, itching, burning, swelling, or a lump under your skin where the needle is placed

If you notice other side effects that you think are caused by this medicine, tell your doctor

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