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Amicus Therapeutics (FOLD)
NASDAQ:FOLD
Migalastat Hcl 20 Mg
Fabry Disease
Phase III
Recruiting
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Mar 13, 2025
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Cipaglucosidase Alfa, Miglustat
Glycogen Storage Disease Type Ii Infantile Onset
Phase III
Recruiting
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Mar 10, 2021
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Atb200, At2221
Pompe Disease (Late-Onset)
Phase III
Completed
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
Oct 18, 2019
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Migalastat Hcl 150 Mg
Fabry Disease
Phase III
Completed
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Jul 30, 2019
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Migalastat Hcl 150 Mg
Fabry Disease
Phase III
Recruiting
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Jun 24, 2019
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Cipaglucosidase Alfa, Miglustat
Pompe Disease (Late-Onset)
Phase III
Recruiting
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Apr 09, 2019
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Cipaglucosidase Alfa, Miglustat, Alglucosidase Alfa
Pompe Disease (Late-Onset)
Phase III
Completed
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
Oct 10, 2018
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Migalastat Hcl 150 Mg
Fabry Disease
Phase III
Completed
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Apr 09, 2018
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At-Gtx-501
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis
Phase I/II
Completed
Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
Mar 16, 2016
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Atb200, At2221
Pompe Disease
Phase I/II
Completed
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Jan 26, 2016
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FAQ
What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases:
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does Amicus Therapeutics (FOLD) have in its pipeline
FOLD is currently developing the following drugs: Migalastat Hcl 20 Mg, Cipaglucosidase Alfa, Miglustat, Atb200, At2221. These drug candidates are in various stages of clinical development as the company works toward FDA approval.