Zolgensma
Generic name:onasemnogene abeparvovec-xioi
Dosage form: intravenous (infusion) injection
Drug class:Miscellaneous uncategorized agents
What is Zolgensma?
Zolgensama is a gene therapy used for spinal muscular atrophy (SMA). SMA is an inherited disorder that causes skeletal muscle weakness and wasting, which worsens with age.
SMA is caused when a gene, usually SMA1, is missing or doesn't work properly. Zolgensama provides a new functional or working copy of a human SMN gene.
Zolgensama is a one-time treatment given via an intravenous (IV) infusion. It was approved by the US Food and Drug Administration (FDA) in 2019.
What is Zolgensma used for?
Zolgensma is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). Zolgensma is given as a one-time infusion into a vein. Zolgensma has not been evaluated in patients with advanced SMA.
Important information
- Zolgensma can increase liver enzyme levels and cause acute serious liver injury or acute liver failure.
- Your child will receive an oral corticosteroid before and after infusion with Zolgensma and will undergo regular blood tests to monitor liver function.
- Contact your child's doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, if they miss a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.
What should I tell my doctor before receiving Zolgensma?
Before receiving Zolgensma, talk with your child's doctor about:
- Vaccinations to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
- Protection against respiratory syncytial virus (RSV), which is recommended.
How should I receive Zolgensma?
- Zolgensma is administered by IV infusion over 60 minutes.
- Zolgensma is administered alongside corticosteroid therapy. Corticosteroid therapy is started one day prior to infusion of Zolgensma and continued for a total of 30 days.
- Contact your child's doctor immediately if your child's skin and/or whites of the eyes appear yellowish, if they miss a dose of corticosteroid or vomits it up, or if the patient experiences a decrease in alertness.
- Before and after your child receives Zolgensma watch for:
- Infections before or after Zolgensma infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see any signs of a possible infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
- Decreased platelet counts could occur following infusion with Zolgensma. Seek immediate medical attention if the patient experiences unexpected bleeding or bruising.
- Thrombotic microangiopathy (TMA) has been reported to occur approximately one week after Zolgensma infusion. Caregivers should seek immediate medical attention if the patient experiences any signs or symptoms o..
