Cipaglucosidase Alfa

Phase 3

Amicus Therapeutics, Inc. (FOLD)

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What is LOA?

Phase 3

Development Phase

Upcoming Catalysts

Historical Events

Regulatory Approvals

Upcoming Catalysts

What are catalysts?

Jul '27

FOLDPhase 3$4.5B

Cipaglucosidase alfa Phase 3 Est. Readout

Cipaglucosidase Alfa• Pompe Disease (Late-onset)36

Jul 2027~ConfirmedSTALE

CT.gov

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Apr '27

FOLDPhase 3$4.5B

Cipaglucosidase alfa Phase 3 Est. Readout

Cipaglucosidase Alfa• Pompe Disease (Late-onset)36

Apr 2027~ConfirmedSTALE

CT.gov

00%

Competitive Landscape

3 competitors in Pompe Disease (Late-onset)

SNYalglucosidase alfa

Phase 3

View Full Landscape

Drug Information

Target Indication

Pompe Disease (Late-onset)

Clinical Trial

NCT03729362

Last updated: 1/2/2026

Patent & Exclusivity

No patent data available. Pre-approval drugs may not have Orange Book listings.

Source: FDA Orange Book

Regulatory Approvals

EMA3/20/2023

Pombiliti

Pombiliti (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (acid ?-glucosidase [GAA] deficiency).

View on EMA

Drug Science

Mechanism of action & safety profile

Developer

FOLD

Amicus Therapeutics, Inc.

Pharmaceutical Preparations•North America•NASDAQ

Related Pipeline

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MiglustatPhase 3

Pompe Disease (Late-onset)

migalastat hydrochloridePhase 3

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