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Phase 3

Development Phase

1

Upcoming Catalysts

0

Historical Events

5

Regulatory Approvals

Upcoming Catalysts
What are catalysts?
Jul '27
FOLDPhase 3

Miglustat Phase 3 Est. Readout

Miglustat
Jul 2027~STALE
CT.gov
00%
Competitive Landscape

3 competitors in Pompe Disease (Late-onset)

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Drug Information

Target Indication

Pompe Disease (Late-onset)

Clinical Trial

NCT03911505

Last updated: 1/8/2026

Patent & Exclusivity
ActiveRLD

Latest Patent Expiry

Sep 16, 2038(12 years)

Patents Listed

10

Exclusivity

NP

Application

NDA 215211
Regulatory Approvals
EMA6/26/2023

Opfolda

Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid ?- glucosidase [GAA] deficiency).

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EMA2/18/2019

Miglustat Dipharma

Miglustat Dipharma is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Dipharma may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Miglustat Dipharma is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

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EMA11/9/2017

Miglustat Gen.Orph

Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Miglustat Gen.Orph is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

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EMA3/22/2017

Yargesa

Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

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EMA11/20/2002

Zavesca

Zavesca is indicated for the oral treatment of adult patients with mild to moderate type-1 Gaucher disease. Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable. Zavesca is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type-C disease.

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Drug Science
Mechanism of action & safety profile
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