Description
Onasemnogene abeparvovec-brve injection is used to treat spinal muscular atrophy (SMA) with confirmed mutation in survival motor neuron 1 (SMN1) gene. Spinal muscular atrophy is a rare neuromuscular disorder that is characterized by loss of lower motor neurons and progressive muscle wasting, often leading to early death.
This medicine is to be given only by or under the direct supervision of your doctor.
This product is available in the following dosage forms:
- Suspension
Before Using
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:
Allergies
Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
Pediatric
Appropriate studies have not been performed on the relationship of age to the effects of onasemnogene abeparvovec-brve injection in children younger than 2 years of age.
Geriatric
No information is available on the relationship of age to the effects of onasemnogene abeparvovec-brve injection in geriatric patients.
Breastfeeding
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Drug Interactions
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. Tell your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicine.
Other Interactions
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.
Other Medical Problems
The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:
- Heart disease or
- Infection, active or
- Liver disease or
- Peripheral neuropathy (nerve problem) or
- Thrombocytopenia (low platelet count)—Use with caution. May make these conditions worse.
Proper Use
A doctor or other trained health professional will give you or your child this medicine in a hospital. This medicine is given through a needle placed into your spinal cord.
Your doctor may give you or your child other medicines (eg, steroids) before and after you receive this medicine to prevent unwanted effects.
Your doctor may also give you or your child a vaccine to prevent respiratory syncytial virus. Talk to your doctor if you have concerns.
Precautions
It is very important that your doctor check you or your child's progress closely while receiving the medicine to make sure it is working properly. Blood and urine tests are needed to check for unwanted effects.
Tell your doctor if you are pregnant. If you can get pregnant, your doctor may do tests to make sure you are not pregnant before starting the treatment. Female patients should not donate egg and must use an effective birth control during treatment with this medicine and for at least 6 months after the last dose. Male patients who have female partners should not donate sperm and must use an effective birth control during treatment with this medicine and for at least 3 months after the last dose.
Check with your doctor right away if you or your child has pain or tenderness in the upper stomach, pale stools, dark urine, loss of appetite, nausea, vomiting, or yellow eyes or skin. These could be symptoms of a serious liver problem.
This medicine may cause you or your child to bruise or bleed more easily. Be extra careful to avoid injuries. Stay away from rough sports or other situations where you could be bruised, cut, or injured. Gently brush and floss your teeth. Be careful when using sharp objects, including razors and fingernail clippers.
This medicine may cause peripheral neuropathy (nerve problem). Check with your doctor right away if you are having burning, numbness, tingling, or painful sensations, weakness in the arms, hands, legs, or feet, or unsteadiness.
Thrombotic microangiopathy (damage in the smallest blood vessels) may occur while you are using this medicine. Tell your doctor right away if you or your child have a decreased urine output, seizures, severe chest pain, sudden weakness in the arms or legs, or unusual bleeding or bruising.
This medicine may cause heart problems. Check with your doctor right away if you or your child have chest pain or discomfort, fast or irregular heartbeat, swelling of the feet and lower legs, or trouble breathing.
This medicine may increase your risk of a tumor. Talk with your doctor if you have concerns about this.
Talk with your doctor before you or your child receive any vaccines during treatment with this medicine. The vaccination schedule should be adjusted as needed to fit in with the steroid treatment schedule.
Side Effects
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.
Check with your doctor or nurse immediately if any of the following side effects occur:
More common
- Arm or leg pain
- black, tarry stools
- bleeding gums
- blood in the urine or stools
- body aches or pain
- burning, numbness, tingling, or painful sensations
- chills
- clay colored stools
- cough
- dark urine
- decreased appetite
- ear congestion
- fever
- headache
- itching, skin rash
- loss of appetite
- loss of voice
- nausea or vomiting
- pinpoint red spots on the skin
- sneezing
- sore throat
- stuffy or runny nose
- swelling of the feet or lower legs
- trouble breathing
- unsteadiness or awkwardness
- unusual bleeding or bruising
- unusual tiredness or weakness
- weakness in the arms, hands, legs, or feet
- yellow eyes or skin
Incidence not known
- Seizures
- sudden, severe chest pain
Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:
More common
- Dizziness
Incidence not known
- Back pain
- chest tightness
- flushing
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.
DRG-80008581
