Onasemnogene abeparvovec (Zolgensma)

Onasemnogene abeparvovec is used to treat spinal muscular atrophy in children under 2 years old who have a specific gene mutation that impacts nerve signals to the muscles.

Onasemnogene abeparvovec may not work in a child who is completely paralyzed or who relies on a ventilator to breathe.

Onasemnogene abeparvovec may also be used for reasons not mentioned in this medication guide.

Your doctor will do blood tests to ensure your child does not have any conditions that would make it unsafe to receive onasemnogene abeparvovec.

Onasemnogene abeparvovec is given as an infusion into a vein, usually just one time. A healthcare provider will give your child this injection.

Onasemnogene abeparvovec must be given slowly, and the infusion can take about 1 hour to finish.

Your child may be given steroid medication to help prevent serious side effects of onasemnogene abeparvovec. Continue to give your child the steroid for as long as your doctor has prescribed. The doctor may gradually lower the dose as your child keeps taking the steroid.

Your child will need regular blood tests to check his or her liver function.

Your child may need to be vaccinated against respiratory syncytial (sin-SISH-ul) virus, or RSV. Follow the doctor's instructions about this vaccination.

If your child vomits shortly after taking the steroid medicine, do not give another dose. Call the doctor for instructions.

onasemnogene abeparvovec can cause serious liver damage, especially in children who already have liver problems. Your doctor will do blood tests to ensure your child does not have conditions that would make it unsafe to receive onasemnogene abeparvovec.

This medicine can cause serious liver damage. Tell your doctor if your child has ever had liver problems.

It may not be safe to breastfeed your child while he or she is receiving this medicine. Ask your doctor about any risks.

Get emergency medical help if your child shows signs of an allergic reaction: hives; difficulty breathing; swelling of the face, lips, tongue, or throat.

Call your doctor right away if your child has:

• jaundice (yellowing of the skin or eyes); or
• easy bruising, unusual bleeding, purple or red spots under the skin.

Common side effects of onasemnogene abeparvovec may include:

• vomiting; or
• abnormal liver function tests.

This is not a complete list of side effects, and others may occur. Call your doctor for medical advice about side effects. You can report side effects to the FDA at 1-800-FDA-1088.

Usual Pediatric Dose for Spinal Muscular Atrophy:

Prior to infusion: -Assess liver function by clinical examination and laboratory testing (ALT, AST, total bilirubin, and prothrombin time)-Obtain creatinine, complete blood count (including hemoglobin and platelet counts), and troponin-I-Assess for the presence of anti-AAV9 antibodiesCorticosteroid -Initiate systemic corticosteroids 1 day prior to IV infusion and continue for a total of 30 days (equivalent to oral prednisolone 1 mg/kg/day) Single-dose: 1.1 x 10(14) vector genomes/kg IV through a venous catheter over 60 minutes Comments:-See Other comments/Administration advice for guidance on administration; the manufacturer's product labeling provides a dose table which includes dose volume (mL) based on weight. -The use in patients with advance spinal muscular atrophy (e.g., complete paralysis of limbs, permanent ventilator-dependence) has not been evaluated. -The safety and effectiveness of repeat administration have not been evaluated. Use: For the treatment of patients less than 2 years with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 gene; because concomitant treatment with corticosteroids may adversely affect neurological development, infusion should be delayed until corresponding full-term gestational age is reached.

Onasemnogene abeparvovec is given as a single dose and does not require daily doses.

Since onasemnogene abeparvovec is given by a healthcare professional in a medical setting, an overdose is unlikely to happen.

If your child is also taking steroid medicine, your child should not receive certain vaccines. If your child needs any vaccine, ask your doctor first before your child gets the shot(s).

Onasemnogene abeparvovec can pass into your child's stools (bowel movements). For at least 1 month after your child gets a dose, avoid letting the child's stools come into contact with hands or other surfaces. Wear rubber gloves while cleaning up the child's body fluids, handling contaminated trash or laundry, or changing diapers. Seal used diapers in disposable bags and throw them away. Wash hands before and after removing gloves. Wash soiled clothing and linens separately from other laundry.

Other drugs may affect onasemnogene abeparvovec, including prescription and over-the-counter medicines, vitamins, and herbal products. Inform your doctor about all your child's current medicines and any medicine the child starts or stops using.