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miglustat

Generic: MIGLUSTAT·Brand: MIGLUSTAT, OPFOLDA, Opfolda, YARGESA, Yargesa, ZAVESCA, Zavesca

miglustat: developed by 1 company · 5 clinical trials tracked · 1 FDA decision.

miglustat evidence translation

What we can confirm

1 FDA record · 5 tracked trials

What it means

Source-backed records connect miglustat to FOLD; that link alone does not prove the drug works.

What to watch

Watch active trials and any change in FDA or trial status.
BioSniper AIPage evidence connected

Prepared research question

How could miglustat → FOLD affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence3 indications · 0 study sponsors

Related indications

Pompe Disease (Late-onset); Pompe DiseasePompe Disease (Late-onset); Glycogen Storage Disease Type II Infantile Onset; Pompe DiseasePompe Disease

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (2)
CompanyPhase
AMICUS THERAPEUTICS, INC. (FOLD)
Pompe Disease (Late-onset); Pompe Disease
Phase 3
AMICUS THERAPEUTICS, INC. (FOLD)
Pompe Disease (Late-onset); Glycogen Storage Disease Type II…
Phase 3
Clinical Trials (5)

A Global Prospective Observational Registry of Patients With Pompe Disease

NCT06121011·FOLD
Recruiting

Primary endpoint: Evaluate long-term safety of Pompe disease treatments

Observational500 targetStarted 2024-02Completion 2034-1241 sites · Austria, Belgium, Denmark, Germany…Amicus Therapeutics

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

NCT04808505·Phase 3·FOLD
Recruiting

Primary endpoint: Proportion of subjects with infusion-associated reactions (IARs)

Interventionalopen36 targetStarted 2023-07Completion 2027-0714 sites · Germany, Italy, Netherlands, Taiwan…Amicus Therapeutics

An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease

NCT03911505·Phase 3·FOLD
Active, not recruiting

Primary endpoint: Incidence of treatment-emergent adverse events (TEAEs) from baseline

Interventionalopen21 targetStarted 2020-02Completion 2026-0617 sites · Australia, Canada, Germany, Italy…Amicus Therapeutics

A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease Compared With Alglucosidase Alfa/Placebo

NCT03729362·Phase 3·FOLD
Completed

Primary endpoint: Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD)

InterventionalRandomizeddouble125 enrolledStarted 2018-12Completion 2021-0150 sites · Argentina, Australia, Austria, Belgium…Amicus Therapeutics

An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

NCT02675465·Phase 1·FOLD
Completed

Primary endpoint: Incidence of Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs), and Adverse Events (AEs) Leading to Discontinuation of Study Drug

Interventionalopen29 enrolledStarted 2016-04Completion 2024-0819 sites · Australia, Germany, Netherlands, New Zealand…Amicus Therapeutics
FDA Decisions1 records
Approval1 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-g(FOLD)FDA ↗
NDA215211Type 5 - New Formulation or New ManufacturerOrphan DrugStandardOralCapsule

Frequently asked questions

Who is developing miglustat?

miglustat is being developed by AMICUS THERAPEUTICS, INC..

How many clinical trials involve miglustat?

BioSniper tracks 5 clinical trials involving miglustat.

What is miglustat's latest FDA decision?

The most recent tracked FDA decision for miglustat is Approval for 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-g, dated September 28, 2023.