Opfolda

Generic name: Miglustat
Dosage form: capsule (65mg)
Drug class: Miscellaneous metabolic agents

Usage of Opfolda

Opfolda (miglustat) is an enzyme stabilizer medicine used in the treatment of late-onset Pompe disease in adults. Opfolda capsules are used in combination with Pombiliti (cipaglucosidase alfa-atga) infusion. The Opfolda capsules are taken to increase the effectiveness of Pombiliti. Opfolda binds to Pombiliti to stabilize and protect it in the bloodstream after infusion. Then once it has been absorbed into the cells Pombiliti becomes unbound so that it can break down excessive glycogen.

Late-onset Pompe disease is a inherited, rare, debilitating, and life-threatening disease caused by a deficiency of the enzyme acid alpha-glucosidase (GAA). Low levels of GAA cause a build up of glycogen in muscle cells which causes muscle damage. Symptoms of late-onset Pompe disease are caused by progressive muscle weakness and include leg and torso weakness, breathing difficulties, sleep apnea, difficulty eating, digestive problems and enlarged heart. Pompe disease may also be called glycogen storage disease, type II, acid maltase deficiency, and glycogenosis type II.

Opfolda side effects

Common Opfolda side effects

The most common side effects affecting 5% or more of patients using Opfolda + Pombiliti in the 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain, and fever.

Less common Opfolda side effects

Other side effects that affected at least 2% of patients treated with Opfolda in combination with Pombiliti across the 3 clinical trials include: muscle pain, joint pain, increased blood pressure, pain, tremor, indigestion, weakness or lack of energy, constipation, infusion site swelling, flank pain, malaise, decreased platelet count, and a burning prickling sensation in parts of the body.

These are not all of the possible side effects of Opfolda in combination with Pombiliti. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Before taking Opfolda

Before taking Opfolda, tell your healthcare provider about all of your medical conditions, including if you:

have any kidney problems

Pregnancy

Tell your healthcare provider if you are pregnant or plan to become pregnant. Opfolda when taken in combination with Pombiliti may cause harm to your unborn baby.

Females who are able to become pregnant:

You should have a pregnancy test to ensure you are not pregnant before you start treatment with Opfolda + Pombiliti.

You should use effective birth control (contraception) during treatment with Opfolda + Pombiliti, and for at least 60 days after the last dose.

If you become pregnant or think you might be pregnant during treatment with Opfolda in combination with Pombiliti you should tell your healthcare provider right away.

Infertility

Advise the male or female of reproductive potential that taking Opfolda with Pombiliti may cause fertility problems, which may affect the ability to have children. Talk to your healthcare provider if you have concerns about fertility.

Breastfeeding

Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if Opfolda alone or in combination with Pombiliti will pass into your breast milk. Do not breastfeed during treatment with Opfolda in combination with Pombiliti. Talk to your healthcare provider about the best way to feed your baby during this time.

Relate drugs

How to use Opfolda

Recommended Adult Opfolda dosage

Dose is based on actual body weight and administered orally every other week.

Dose:

260 mg for patients weighing ≥50 kg.

195 mg for patients weighing ≥40 kg to <50 kg.

Comment: See full prescribing information for recommended dosage in patients with renal impairment.

Opfolda + Pombiliti should be used together.

Warnings

Do not take Opfolda in combination with Pombiliti if you are pregnant.

Opfolda must be administered in combination with Pombiliti. Refer to the Pombiliti Prescribing Information for a description of additional risks for Pombiliti including, but not limited to, the warnings and precautions for Pombiliti.

What other drugs will affect Opfolda

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Disclaimer

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