Description
Pariglasgene brecaparvovec-opnr injection is used with proper diet to help reduce the amount of cornstarch taken daily in patients with glycogen storage disease type Ia (GSDIa). Pariglasgene brecaparvovec-opnr is an adeno-associated virus (AAV) vector-based gene treatment that works by copying the gene needed for the human G6PC promoter.
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 performed to date have not demonstrated pediatric-specific problems that would limit the usefulness of pariglasgene brecaparvovec-opnr injection in children 8 years of age and older. However, safety and efficacy have not been established in children younger than 8 years of age.
Geriatric
Appropriate studies on the relationship of age to the effects of pariglasgene brecaparvovec-opnr injection have not been performed in the elderly.
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:
- Adrenal gland problem or
- Liver disease—Use with caution. May make these conditions worse.
- Liver disease (eg, hepatic fibrosis, cirrhosis), severe—Should not be used in patients with this condition.
Proper Use
A doctor or other trained health professional will give you this medicine. It is given through an IV catheter that is placed into one of your veins. It must be given slowly, so the IV will have to remain in place for at least 60 to 180 minutes.
Your doctor will give you other medicines (eg, allergy, fever, or steroid medicine) before you receive this medicine to help prevent unwanted effects.
This medicine is a virus vector-based gene treatment and may spread to other people through urine, stool, and saliva. Wash your hands with soap and water after handling the patient's body waste. Follow these precautions during treatment and for 3 months after the infusion.
Precautions
It is very important that your doctor check your or your child's progress closely and at regular visits to make sure this medicine is working properly and to decide if you should continue to receive it. Blood and urine tests may be needed to check for unwanted effects.
Receiving this medicine while you are pregnant can harm your unborn baby. It may also cause birth defects if the father is receiving it when his sexual partner becomes pregnant. Female patients should use effective birth control during treatment with this medicine and for at least 12 months after the last dose. Male patients who have female partners should use effective birth control during treatment with this medicine and for at least 6 months after the last dose. If you think you have become pregnant while receiving the medicine, tell your doctor right away.
Male patients should not donate sperm during treatment with this medicine and for at least 6 months after the last dose.
This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you have blurred vision, chest discomfort or tightness, confusion, dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position, fast heartbeat, flushing, redness of the skin, rash, itching, hoarseness, sweating, trouble breathing, trouble swallowing, unusually warm skin, any swelling of your hands, face, or mouth, or unusual tiredness or weakness after receiving this medicine.
This medicine may cause infusion reactions, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you start to have chest discomfort, fever, chills or shaking, confusion, difficulty swallowing, dizziness, fast heartbeat, flushing, redness of the skin, increase or decrease blood pressure, trouble breathing, itching or rash, unusually warm skin, or lightheadedness, or fainting after receiving this medicine.
Check with your doctor right away if you have clay colored stools, dark urine, decreased appetite, fever, headache, loss of appetite, nausea and vomiting, skin rash or itching, stomach pain or tenderness, swelling of the feet or lower legs, unusual tiredness or weakness, or yellow eyes or skin. These may be symptoms of serious liver problems.
This medicine may cause adrenal gland problems. Check with your doctor right away if you have darkening of the skin, diarrhea, dizziness, fainting, loss of appetite, mental depression, nausea, skin rash, unusual tiredness or weakness, or vomiting.
This medicine may increase your risk of a tumor. Talk with your doctor if you have concerns about this.
Talk to your doctor before receiving vaccines during treatment this medicine. Do not receive vaccines for 1 month before treatment with this medicine.
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
- Blurred vision
- chest tightness
- confusion
- cough
- diarrhea
- difficulty swallowing
- dizziness
- dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position
- dry mouth
- fast or slow heartbeat
- feeling of warmth
- fever
- flushed, dry skin
- fruit-like breath odor
- general feeling of discomfort or illness
- headache
- hives, itching, skin rash
- increased fat deposits on the face, neck, and trunk
- increased hunger
- increased thirst
- increased urination
- joint pain
- large amount of triglyceride in the blood
- loss of appetite
- muscle aches and pains
- nausea
- nervousness
- pounding in the ears
- puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue
- redness of the face, neck, arms and occasionally, upper chest
- runny nose
- shivering
- sore throat
- stomach pain
- sweating
- trouble breathing
- trouble sleeping
- unexplained weight loss
- unusual tiredness or weakness
- vomiting
Less common
- Anxiety
- chills
- cold sweats
- coma
- cool, pale skin
- darkening of the skin
- depression
- nightmares
- seizures
- shakiness
- slurred speech
- swelling of the feet or lower legs
- yellow eyes or skin
Incidence not known
- Clay colored stools
- dark urine
- facial swelling
- muscle cramps
- stomach tenderness
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
- Blemishes on the skin
- constipation
- pimples
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-80012060
