What is Onasemnogene Abeparvovec-xioi and where it is used?
This is a gene replacement therapy used for the treatment of spinal muscular atrophy (SMA) in pediatric patients. The Food and Drug Administration (FDA) has approved this medication in 2019.
SMA is a rare genetic disease that deteriorates the neuromuscular functioning of the body. It is by causing motor neurons loss and associated muscle weakness and paralysis. The disease is caused by a genetic defect in the survival of motor neurons. SMA is categorized into SMA Type 1 and SMA type 2. The first form is a more severe variant that causes rapid motor neuron loss and resulting in death if left untreated.
How does Onasemnogene Abeparvovec-xioi work?
This drug targets the genetic root cause of SMA. It is by replacing the function of the missing or nonworking gene called SMN1gene. This gene is critical to making SMN protein. SMN protein is essential to motor neuron cell survival. This medicine is made up of a new working copy of a human SMN gene that is placed inside a vector. The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9. This type of virus does not make people sick. Vectors are used because they can travel throughout the body and deliver the new working gene to the cells where it is needed.
When the new gene reaches its destination, it is ready to tell the motor neuron cells to start making SMN protein. With the motor neuron cells now able to make sufficient SMN protein, motor neuron cells that have not died anymore may survive. They may also function and be maintained.
How to use this medicine?
Patients will receive an oral corticosteroid before and after infusion with this medication. They will also undergo regular blood tests to monitor liver function. Follow your doctor’s orders. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so. The amount of medicine that you take depends on the strength of the medicine. Similarly, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.
What are the side effects?
Common side effects:
- Abnormal liver function tests
Call your doctor right away if you have or your child has these side effects:
- Easy bruising
- Unusual bleeding
- Purple or red spots under your skin
- Yellowing of the skin or eyes (jaundice)
Get emergency medical help if your child has signs of an allergic reaction:
- Swelling of the lips, tongue, face, or throat
- Difficulty breathing
- This drug can cause acute serious liver injury. The liver enzymes in children who receive Onasemnogene Abeparvovec-xioi may become elevated and may reflect acute serious liver injury.
- Viral respiratory infections before or after Onasemnogene Abeparvovec-xioi infusion can lead to more serious complications. Contact the patient’s doctor instantly if you see signs of a possible viral respiratory infection. Some of the symptoms may include sore throat, coughing, sneezing, wheezing, runny nose, or fever.
- Decreased platelet counts could occur following infusion with Onasemnogene Abeparvovec-xioi. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.
- Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.