Pariglasgene brecaparvovec

Pariglasgene brecaparvovec is a gene therapy that is used together with dietary treatment to help reduce daily cornstarch intake in adults and children 8 years and older who have glycogen storage disease type Ia (GSDIa).

Pariglasgene brecaparvovec may also be used for reasons not mentioned in this medication guide.

Your doctor will do medical tests before starting treatment with pariglasgene brecaparvovec to ensure it is the right treatment for you.

Pariglasgene brecaparvovec is given in one dose as an infusion into a vein. A healthcare provider will administer this infusion.

You will receive other medicines before your infusion to help lower the risk of infusion reactions, including allergic reactions.

You will also need to take a corticosteroid medicine starting 2 weeks after your infusion to help reduce the risk of liver problems. Take it exactly as prescribed for as long as your doctor advises. Do not stop taking it suddenly.

You will need blood tests before and after receiving this medicine to check your liver and adrenal gland function.

Pariglasgene brecaparvovec genetic material may be found in your blood, urine, stool, and saliva after treatment. Follow your healthcare provider's instructions for careful hygiene when handling body waste for 3 months after your infusion.

You should not be treated with pariglasgene brecaparvovec if you have severe liver fibrosis or cirrhosis.

You should not be treated with pariglasgene brecaparvovec if you have severe liver fibrosis or cirrhosis.

Tell your doctor if you have or have ever had:

• an allergic reaction to pariglasgene brecaparvovec; or
• liver problems or an acute liver infection.

Tell your doctor if you have recently received or are scheduled to receive a vaccine. You should not receive vaccines for 1 month before receiving this medicine. Your doctor may need to change your vaccination schedule.

Using pariglasgene brecaparvovec may increase your risk of developing a tumor. Ask your doctor about your individual risk.

Pariglasgene brecaparvovec should not be used during pregnancy. It is not known if this medicine will harm an unborn baby.

• If you are a woman who can become pregnant, use birth control while using this medicine and for at least 12 months after your last dose.
• If you are a man, use birth control if your sex partner is able to get pregnant. Keep using birth control for at least 6 months after your last dose.
• Tell your doctor right away if you become pregnant or think you are pregnant.

Men using pariglasgene brecaparvovec should not donate semen for at least 6 months after treatment.

Ask your doctor if it is safe to breastfeed while using this medicine.

Get emergency medical help if you have signs of an allergic reaction: hives, trouble breathing, swelling of your face, lips, tongue, or throat.

Some side effects may happen during the infusion. Tell your medical caregiver if you feel dizzy, nauseated, light-headed, itchy, sweaty, or have a headache, chest tightness, back pain, difficulty breathing, or swelling in your face.

Pariglasgene brecaparvovec may cause serious side effects. Call your doctor right away if you have:

• a tumor (an abnormal growth of tissue);
• liver problems--loss of appetite, nausea, vomiting, stomach pain (upper right side), tiredness, itching, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes); or
• decreased adrenal gland hormones--feeling light-headed as if you might faint, muscle or joint pain, loss of consciousness, skin discoloration, craving salty foods.

Common side effects of pariglasgene brecaparvovec may include:

• abnormal liver function tests; or
• nausea.

This is not a complete list of side effects and others may happen. Call your doctor for medical advice about side effects. You can report side effects to the FDA at 1-800-FDA-1088.

Usual Adult Dose for Glycogen Storage Disease Type Ia:

1 x 10(13) genome copies/kg IV as a single infusionTotal dose volume based on body weight range:-Weight 15 to less than 21 kg: 6 mL-Weight 21 to less than 27 kg: 8 mL-Weight 27 to less than 33 kg: 10 mL-Weight 33 to less than 39 kg: 12 mL-Weight 39 to less than 45 kg: 14 mL-Weight 45 to less than 51 kg: 16 mL-Weight 51 to less than 57 kg: 18 mL-Weight 57 to less than 63 kg: 20 mL-Weight 63 to less than 69 kg: 22 mL-Weight 69 to less than 75 kg: 24 mL-Weight 75 to less than 81 kg: 26 mL-Weight 81 to less than 87 kg: 28 mL-Weight at least 87 kg: 30 mLComments:-Dose volume is rounded up or down to the nearest 2 mL.-This drug has not been studied in patients 65 years and older.Use: To reduce daily cornstarch intake as an adjunct to nutritional management in patients with glycogen storage disease type Ia

Usual Pediatric Dose for Glycogen Storage Disease Type Ia:

8 years and older: 1 x 10(13) genome copies/kg IV as a single infusionTotal dose volume based on body weight range:-Weight 15 to less than 21 kg: 6 mL-Weight 21 to less than 27 kg: 8 mL-Weight 27 to less than 33 kg: 10 mL-Weight 33 to less than 39 kg: 12 mL-Weight 39 to less than 45 kg: 14 mL-Weight 45 to less than 51 kg: 16 mL-Weight 51 to less than 57 kg: 18 mL-Weight 57 to less than 63 kg: 20 mL-Weight 63 to less than 69 kg: 22 mL-Weight 69 to less than 75 kg: 24 mL-Weight 75 to less than 81 kg: 26 mL-Weight 81 to less than 87 kg: 28 mL-Weight at least 87 kg: 30 mLComments:-Dose volume is rounded up or down to the nearest 2 mL.Use: To reduce daily cornstarch intake as an adjunct to nutritional management in patients with glycogen storage disease type Ia

Pariglasgene brecaparvovec is given as a single dose. If you miss an appointment for your pariglasgene brecaparvovec infusion, call your doctor for instructions.

An overdose of this product is not likely to happen. However, if it does occur in a medical setting, it will be treated quickly.

Drinking alcohol may raise your risk of liver damage.

Do not take any medicine, vitamin, herbal product, or nutritional supplement without your doctor's advice.

Other drugs may influence pariglasgene brecaparvovec, including prescription medicines, over-the-counter medicines, vitamins, and herbal products. Inform your doctor about all other medicines you are using.