Onasemnogene abeparvovec-xioi (Intravenous)

Onasemnogene abeparvovec-xioi injection is used to treat spinal muscular atrophy (SMA) caused by bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. Spinal muscular atrophy is a rare disorder that affects the nerves controlling muscles, leading to muscle weakness and loss, which can result in early death. Onasemnogene abeparvovec-xioi is a gene therapy that uses an adeno-associated virus to provide the gene needed for making the human SMN protein.

This medicine must be given only by your doctor or under their direct supervision.

It is very important that your doctor monitors your child's progress closely while they are on this medicine to ensure it is working properly. Blood and urine tests are necessary to check for side effects.

Contact your doctor immediately if your child experiences pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, vomiting, or yellowing of the eyes or skin. These could be signs of a serious liver issue.

This medicine may lead to heart problems. Reach out to your doctor right away if your child has chest pain or discomfort, a fast or irregular heartbeat, swelling in the feet and lower legs, or difficulty breathing.

This medicine may make your child more prone to bruising or bleeding. Be extra cautious to avoid injuries. Avoid rough sports or situations where injuries could happen. Gently brush and floss your teeth, and be careful when using sharp objects, like razors and fingernail clippers.

Thrombotic microangiopathy (damage in the smallest blood vessels) may happen while using this medicine. Inform your doctor immediately if your child has a decreased urine output, seizures, severe chest pain, sudden weakness in the arms or legs, or unusual bleeding or bruising.

Discuss with your doctor before your child gets any vaccines during treatment with this medicine. The vaccination schedule may need to be adjusted to align with the steroid treatment schedule.

This medicine may increase your risk of a tumor. Talk to your doctor if you have any concerns about this.

In deciding to use a medicine, you and your doctor need to consider the risks of taking the medicine against the benefits it may provide. For this medicine, the following points should be thought about:

Allergies

Inform your doctor if you have ever had any unusual or allergic reactions to this medicine or any other medicines. Also, let your healthcare provider know if you have any other allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, carefully read the label or package ingredients.

Pediatric

Studies done so far have not shown any specific problems in children from newborns to those younger than 2 years that would limit the usefulness of onasemnogene abeparvovec-xioi injection. However, it is not recommended for premature infants. Its safety and effectiveness have been confirmed.

Geriatric

There is no information available about how age affects the effects of onasemnogene abeparvovec-xioi injection in older patients.

Breast Feeding

There are no adequate studies in women to determine the risk to infants when using this medication during breastfeeding. Consider the potential benefits and risks before taking this medication while breastfeeding.

Interactions with Medicines

Some medicines should not be used together, while others may be used together even if there is a chance of interaction. In these cases, your doctor may adjust the dose or take other precautions. Inform your healthcare provider if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicines.

Interactions with Food/Tobacco/Alcohol

Some medicines should not be taken around mealtime or with certain types of food due to possible interactions. Using alcohol or tobacco with certain medicines may also lead to interactions. Discuss with your healthcare provider about using your medicine with food, alcohol, or tobacco.

Other Medical Problems

Having other medical issues may affect how this medicine is used. Be sure to tell your doctor if you have any other medical conditions, especially:

• Heart disease or
• Active infection or
• Liver disease or
• Thrombocytopenia (low platelet count)—Use with caution. This may worsen these conditions.

Along with its necessary effects, a medicine may cause some unwanted effects. Not all of these side effects may happen, but if they do, they may require medical attention.

Check with your doctor or nurse immediately if any of the following side effects occur:

More common

• Clay-colored stools
• Dark urine
• Decreased appetite
• Fever
• Headache
• Itching, skin rash
• Light-colored stools
• Loss of appetite
• Nausea
• Stomach pain or tenderness
• Swelling of the feet or lower legs
• Unusual tiredness or weakness
• Vomiting
• Yellow eyes or skin

Incidence not known

• Back pain
• Black, tarry stools
• Bleeding gums
• Blood in the urine or stools
• Blurred vision
• Confusion
• Decreased urine output
• Dizziness
• Dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position
• Drowsiness
• Pinpoint red spots on the skin
• Seizures
• Sudden weakness in the arms or legs
• Sudden, severe chest pain
• Sweating
• Unusual bleeding or bruising

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.

In the U.S.

• Zolgensma

Available Dosage Forms:

• Suspension

Therapeutic Class: Musculoskeletal Agent

Find detailed information on biosimilars for this medication.

A nurse or other trained health professional will give your child this medicine in a medical facility. It is given through a needle placed into one of your child's veins. The medicine must be given slowly, so the needle will have to stay in place for at least 60 minutes.

Your doctor may give your child other medicines (eg, steroids) before and after the infusion to prevent unwanted effects.

Your doctor may also give your child a vaccine to prevent respiratory syncytial virus. Talk to your doctor if you have concerns.

This medicine is a virus vector-based gene treatment and may spread to other people through body waste (feces). Seal diapers in trash bags and throw them into the trash. Wash your hands with soap and water before and after handling your child's body waste. Follow these precautions during treatment with this medicine and for 1 month after the infusion.

• Is Zolgensma a cure for SMA?