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🏅 FDA Orphan Designation

Pombiliti and Opfolda

cipaglucosidase alfa-atga and miglustat

Manufacturer: Amicus Therapeutics, Inc.

Indicated for:
Glycogen storage disease due to acid maltase deficiencyOrphan

FDA-Approved Indications (1)

in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing greater than or equal to 40 kg and who are…

Indications & Usage

in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing greater than or equal to 40 kg and who are not improving on their current enzyme replacement therapy (ERT) ||| in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing greater than or equal to 40 kg and who are not improving on their current enzyme replacement therapy (ERT)

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Pombiliti and Opfolda
Amicus Therapeutics, Inc.
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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.