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Opfolda Coupon - Opfolda 65mg capsule

Opfolda

miglustat
Used for Lysosomal Storage Disease
Used for Lysosomal Storage Disease

Opfolda (miglustat) is an enzyme stabilizer. It's used along with another medication called Pombiliti (cipaglucosidase alfa-atga) to treat Pompe disease in adults. Opfolda (miglustat) protects Pombiliti (cipaglucosidase alfa-atga) from being broken down in the body and is taken by mouth an hour before a Pombiliti (cipaglucosidase alfa-atga) infusion. You'll typically receive this treatment every 2 weeks, as directed by your provider. The most common side effects of this combination are headache and diarrhea.

Reviewed by:
reviewer
Robert Chad Hakim, PharmD, BCCCP
reviewer
Kristianne Hannemann, PharmD
Last reviewed on January 18, 2024
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What is Opfolda (miglustat)?

What is Opfolda (miglustat) used for?

How Opfolda (miglustat) works

People who have Pompe disease don't have enough of an enzyme (protein) called acid alpha-glucosidase (GAA). This enzyme typically helps convert glycogen stored in cells into sugars to be used for energy. Without enough GAA, glycogen builds up in the cells and can cause problems, such as muscle weakness.

Pombiliti (cipaglucosidase alfa-atga) is an enzyme replacement therapy (ERT) used to replace GAA in people with Pompe disease. Opfolda (miglustat) is an enzyme stabilizer. It attaches to Pombiliti to prevent it from breaking down in the body. This allows Pombiliti enough time to get absorbed into the cells, where it can do its job of breaking down glycogen.

Are you looking for information on Galafold (migalastat) instead?

Drug Facts

Common BrandsOpfolda
Drug ClassEnzyme stabilizer
Controlled Substance ClassificationNot a controlled medication
Generic StatusNo lower-cost generic available
AvailabilityPrescription only
faqs

Frequently asked questions about Opfolda (miglustat)

What is Pompe disease?
Pompe disease is a genetic condition. People with this condition don't have enough of an enzyme (protein) called acid alpha-glucosidase (GAA). Normally, this enzyme provides energy to your cells by breaking down the stored form of sugar called glycogen. Without GAA, the glycogen can't be broken down, which can cause muscle weakness. While Pompe disease can affect your whole body, your heart and skeletal muscles are the most affected, which can be life-threatening.
How is Opfolda (miglustat) administered?
Opfolda (miglustat) is available as capsules. Your provider will determine your dose based on body weight and if you have kidney problems. Opfolda (miglustat) is taken by mouth once every 2 weeks on an empty stomach, an hour before the Pombiliti (cipaglucosidase alfa-atga) infusion. You won't be able to eat or drink anything 2 hours before and 2 hours after taking this medication]. But you can take Opfolda (miglustat) with an unsweetened drink, like water or tea or coffee with no cream, sugar, or other sweeteners.
Is Opfolda (miglustat) safe during pregnancy?
No, Opfolda (miglustat) and Pombiliti (cipaglucosidase alfa-atga) must be avoided during pregnancy. Studies suggest this combination can cause severe harm to babies if taken by mothers during pregnancy. If you're able to become pregnant, your provider will ask you to take a pregnancy test before starting treatment. In addition, you should use birth control during and for at least 2 months after the last dose of Opfolda and Pombiliti. Talk to your provider about your options if you're thinking about getting pregnant or are pregnant.
What happens if I miss a Opfolda (miglustat) dose?
If you missed your Opfolda (miglustat) dose, your Pombiliti (cipaglucosidase alfa-atga) infusion will need to be rescheduled. It's very important to not miss any doses because doing so can cause your Pompe disease symptoms to worsen or come back. Mark the days and times of when you need to see your provider and receive Pombiliti (cipaglucosidase alfa-atga) so you know when to stop eating and drinking and take Opfolda (miglustat). If you miss a Opfolda (miglustat) dose, call your provider right away to reschedule your treatment.
What are some side effects of Opfolda (miglustat)?
In clinical studies, people taking Opfolda (miglustat) with Pombiliti (cipaglucosidase alfa-atga) most commonly reported headache and diarrhea. Other less common side effects include dizziness, shortness of breath, bloating, fever, and rash.
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alternatives

What are alternatives to Opfolda (miglustat)?

There are a number of medications that your doctor can prescribe in place of Opfolda (miglustat). Compare a few possible alternatives below.
Opfolda (miglustat)
Used for:
View Prices
Nexviazyme (avalglucosidase alfa)
Used for:

  • Late-onset Pompe disease (LOPD)

View Prices
Lumizyme (alglucosidase alfa)
Used for:

  • Pompe disease

View Prices
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Opfolda (miglustat) images

White And Gray At2221 - OPFOLDA 65mg Capsule
This medicine is White And Gray Capsule Imprinted With "At2221".White And Gray At2221 - OPFOLDA 65mg Capsule
White And Gray At2221 - OPFOLDA 65mg Capsule
This medicine is White And Gray Capsule Imprinted With "At2221".White And Gray At2221 - OPFOLDA 65mg Capsule
White And Gray At2221 - OPFOLDA 65mg Capsule
This medicine is White And Gray Capsule Imprinted With "At2221".White And Gray At2221 - OPFOLDA 65mg Capsule

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References

Best studies we found

Amicus Therapeutics US, LLC. (2023). Opfolda- miglustat capsule [package insert]. DailyMed.

Fukuda, T., et al. (2007). Acid alpha-glucosidase deficiency (Pompe disease). Current Neurology and Neuroscience Reports.

MedlinePlus. (2010). GAA gene.

View All References (2)

National Institute of Neurological Disorders and Stroke. (2023). Pompe disease.

National Organization for Rare Disorders. (2024). Pompe disease.

GoodRx Health has strict sourcing policies and relies on primary sources such as medical organizations, governmental agencies, academic institutions, and peer-reviewed scientific journals. Learn more about how we ensure our content is accurate, thorough, and unbiased by reading our editorial guidelines.
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