Onasemnogene Abeparvovec suspension for injection

What is this medication?

ONASEMNOGENE ABEPARVOVEC (ON a SEM noe jeen A be PAR voe vek) is used to treat patients with spinal muscular atrophy (SMA). It is not a cure.

This medicine may be used for other purposes; ask your health care provider or pharmacist if you have questions.

COMMON BRAND NAME(S): ZOLGENSMA, ZOLGENSMA Suspension

What should I tell my care team before I take this medication?

They need to know if you have any of these conditions:

• heart disease
• liver disease
• low blood counts, like a low platelet count
• an unusual or allergic reaction to onasemnogene abeparvovec, other medicines, foods, dyes or preservatives
• pregnant or trying to get pregnant
• breast-feeding

How should I use this medication?

This medicine is for infusion into a vein. It is given by a health care professional in a hospital or clinic setting.

Talk to your pediatrician regarding the use of this medicine in children. While this drug may be prescribed for children as young as newborns for selected conditions, precautions do apply.

Overdosage: If you think you have taken too much of this medicine contact a poison control center or emergency room at once.

NOTE: This medicine is only for you. Do not share this medicine with others.

What if I miss a dose?

This does not apply. This medicine is not for regular use.

What may interact with this medication?

Interactions are not expected.

This list may not describe all possible interactions. Give your health care provider a list of all the medicines, herbs, non-prescription drugs, or dietary supplements you use. Also tell them if you smoke, drink alcohol, or use illegal drugs. Some items may interact with your medicine.

What should I watch for while using this medication?

Your condition will be monitored carefully while you are receiving this medicine. You will need blood work done while you are taking this medicine.

This medicine may be present in urine and stool. Special care for handling body waste, trash, laundry, spills, and family interactions may be needed. Talk to your healthcare professional about how to protect yourself and family.

What side effects may I notice from receiving this medication?

Side effects that you should report to your doctor or health care professional as soon as possible:

• allergic reactions like skin rash, itching or hives; swelling of the face, lips, or tongue
• signs and symptoms of infection like fever; chills; cough; sore throat
• signs and symptoms of liver injury like dark yellow or brown urine; general ill feeling or flu-like symptoms; light-colored stools; loss of appetite; nausea; right upper belly pain; unusually weak or tired; yellowing of the eyes or skin
• unusual bruising or bleeding

Side effects that usually do not require medical attention (report these to your doctor or health care professional if they continue or are bothersome):

• vomiting

This list may not describe all possible side effects. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Where should I keep my medication?

This medicine is given in a hospital or clinic and will not be stored at home.

NOTE: This sheet is a summary. It may not cover all possible information. If you have questions about this medicine, talk to your doctor, pharmacist, or health care provider.