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🏅 FDA Orphan Designation

OPFOLDA

MIGLUSTAT

Manufacturer: AMICUS THERAPEUTICS US, LLC

Indicated for:📚 Gaucher disease

Indications & Usage

1 INDICATIONS AND USAGE OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). OPFOLDA is an enzyme stabilizer indicated, in combination with Pombiliti, a hydrolytic lysosomal glycogen-specific enzyme, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). ( 1 )

💙 Support Programs

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Pombiliti and Opfolda
Amicus Therapeutics, Inc.
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Miglustat
Actelion
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OPFOLDA
Amicus
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Pombiliti and Opfolda
Amicus Therapeutics, Inc.

Where shown, WAC (Wholesale Acquisition Cost) is an estimate only — your actual cost depends on your insurance plan. Always verify pricing with your pharmacy or insurer.

Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.