Cipaglucosidase alfa

Cipaglucosidase alfa is used along with Opfolda (miglustat) to treat late-onset Pompe disease in adults who weigh at least 88 pounds (40 kg), and when enzyme replacement therapy has not been effective or has stopped being effective.

Cipaglucosidase alfa may also be used for other reasons not mentioned in this medication guide.

Follow all directions on your prescription label and read all medication guides or instruction sheets. Cipaglucosidase alfa must be given together with Opfolda (miglustat).

Your doctor will stop your other treatments for Pompe disease before your first dose of cipaglucosidase alfa. Follow your doctor's dosing instructions very carefully.

Make sure you take Opfolda (miglustat) 1 hour before receiving cipaglucosidase alfa.

You will receive cipaglucosidase alfa as an infusion into a vein about 1 hour after you take Opfolda (miglustat). A healthcare provider will give you this injection every other week.

Your breathing, blood pressure, oxygen levels, and other vital signs will be monitored closely to ensure you do not have serious side effects.

You may be given other medications to help prevent or treat serious side effects or allergic reactions. Continue taking these medicines for as long as your doctor has prescribed.

Doses are based on weight. Your dose may change if you gain or lose weight.

Cipaglucosidase alfa must be given together with Opfolda (miglustat).

Some people can have a severe reaction to cipaglucosidase alfa. Inform your medical caregiver if you have a skin rash or hives, chills, swelling, feel faint, chest pain, trouble breathing, or severe dizziness.

Individuals with serious heart or lung issues, or those prone to fluid retention, may have a higher risk of their condition getting worse. You will be monitored closely to ensure you do not experience serious side effects.

Tell your doctor if you have or have ever had:

• fluid retention;
• heart problems; or
• lung disease or breathing problems.

People with severe heart or breathing problems or those who tend to retain fluid may have a higher risk of their condition getting worse. Discuss your risk with your doctor.

May harm an unborn baby. Do not use if you are pregnant. You will need to have a negative pregnancy test before starting this treatment. Use effective birth control while receiving this treatment and for at least 60 days after your last dose. Inform your doctor if you become pregnant.

Do not breastfeed while receiving cipaglucosidase alfa.

When used together with Opfolda (miglustat), cipaglucosidase alfa may affect fertility in men or women. It may be harder to get pregnant while either parent is using this medicine.

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

Some people have a severe reaction to cipaglucosidase alfa. These side effects may occur during or within a few hours after the injection. Tell your medical caregiver if you feel dizzy, nauseated, itchy, sweaty, light-headed, have a headache, chest pain or tightness, back pain, chills, swelling, or trouble breathing.

Also tell your medical caregivers right away if you have:

• slow heart rate, weak pulse, fainting, slow breathing (breathing may stop).

Common side effects of cipaglucosidase alfa may include:

• headache, weak or tired feeling;
• diarrhea, nausea, stomach pain; or
• fever.

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

Usual Adult Dose for Pompe disease:

At least 40 kg: 20 mg/kg IV every other week, infused over approximately 4 hours -This drug must be given in combination with miglustat (Opfolda), an enzyme stabilizer. Comments:-Therapy with this drug may be started 2 weeks after the last dose of enzyme replacement therapy (ERT). -Calculate the dose of this drug using the patient's actual body weight. -Miglustat should be orally administered 1 hour before initiating infusion of this drug; refer to the manufacturer product information for miglustat for dosage and administration timeline instructions. Use: In combination with miglustat, for the treatment of late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) in patients weighing 40 kg or greater and who are not improving on their current ERT.

You must take Opfolda (miglustat) before getting your cipaglucosidase alfa injection. Contact your doctor for guidance if you miss a dose of Opfolda or an appointment for the injection.

In a medical setting, an overdose would be handled promptly.

Follow your doctor's instructions regarding any limits on food, drinks, or activities.

Other drugs may affect cipaglucosidase alfa, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all other medicines you use.