Amicus Therapeutics

🇺🇸United States

Country: 🇺🇸United States

Ownership: Public

Established: 2002-01-01

Employees: 517

Market Cap: $3.3B

Website: http://www.amicusrx.com

Introduction: Amicus Therapeutics, Inc. operates as a biotechnology company. The firm focuses on discovering, developing and delivering medicines for people living with metabolic diseases. Its product portfolio includes first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants, a novel, clinical-stage, treatment paradigm for Pompe disease and a rare disease gene therapy portfolio. The company was founded by John Francis Crowley on February 4, 2002 and is headquartered in Princeton, NJ.

Clinical Trials

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

Phase 1

Completed

Conditions: Variant Late-Infantile Neuronal Ceroid Lipofuscinosis

Interventions: Genetic: AT-GTX-501

First Posted Date: 2016-04-01

Last Posted Date: 2023-03-08

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 13

Registration Number: NCT02725580

Locations: 🇺🇸
Nationwide Children's Hosptial, Columbus, Ohio, United States

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Phase 1

Completed

Conditions: Pompe Disease

Interventions: Drug: ATB200
Drug: AT2221

First Posted Date: 2016-02-05

Last Posted Date: 2024-08-23

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 32

Registration Number: NCT02675465

Locations: 🇺🇸
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), Fairfax, Virginia, United States
🇺🇸
Emory University Division of Medical Genetics, Decatur, Georgia, United States
🇬🇧
University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center, Birmingham, United Kingdom

and more 16 locations

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

Phase 3

Completed

Conditions: Fabry Disease

Interventions: Drug: migalastat HCl 150 mg

First Posted Date: 2014-07-21

Last Posted Date: 2020-12-21

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 84

Registration Number: NCT02194985

Locations: 🇬🇧
Clinical Study Site, London, United Kingdom

A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers

Phase 1

Completed

Conditions: Fabry Disease

Interventions: Drug: IV migalastat HCl
Drug: IV placebo
Drug: oral migalastat HCl

First Posted Date: 2014-03-10

Last Posted Date: 2014-06-18

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 31

Registration Number: NCT02082327

Locations: 🇳🇱
PRA International, Groningen, Netherlands

A Phase I, Randomized, Single-Blind, Four-Period Cross-Over, Placebo-Controlled, Dose-Escalation Study to Evaluate the Safety and Pharmacokinetics of Single Oral Doses of GR181413A/AT1001 in Healthy Japanese Subjects

Phase 1

Completed

Conditions: Fabry Disease

Interventions: Drug: GR181413A/AT1001 capsule
Drug: GR181413A/AT1001 solution
Other: Potable water
Drug: Placebo capsule

First Posted Date: 2013-05-15

Last Posted Date: 2013-12-18

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 14

Registration Number: NCT01853852

Locations: 🇦🇺
GSK Investigational Site, Randwick, New South Wales, Australia

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

Phase 1

Completed

Conditions: Fabry Disease

Interventions: Drug: AT1001 150 mg

First Posted Date: 2012-11-21

Last Posted Date: 2017-08-03

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 32

Registration Number: NCT01730469

Locations: 🇺🇸
GSK Investigational Site, Orlando, Florida, United States

A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)

Phase 1

Completed

Conditions: Fabry Disease

Interventions: Drug: [14C] AT1001

First Posted Date: 2012-11-21

Last Posted Date: 2013-12-18

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 6

Registration Number: NCT01730482

Migalastat Food Effect Study

Phase 1

Completed

Conditions: Fabry Disease

Interventions: Drug: A (migalastat)
Drug: B (migalastat)
Drug: C (migalastat)
Drug: D (migalastat)
Drug: E (migalastat)

First Posted Date: 2011-12-12

Last Posted Date: 2013-12-18

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 20

Registration Number: NCT01489995

Locations: 🇺🇸
GSK Investigational Site, Austin, Texas, United States

Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease

Conditions: Fabry Disease

First Posted Date: 2011-11-22

Last Posted Date: 2025-05-07

Lead Sponsor: Amicus Therapeutics

Registration Number: NCT01476163

Locations: 🇺🇸
CHOC Children's Hospital Division of Metabolic Disease, Orange, California, United States
🇺🇸
University of Maryland Medical Center, Baltimore, Maryland, United States
🇺🇸
Kidney Care and Transplant Services of New England, Springfield, Massachusetts, United States

and more 2 locations

Open-Label Phase 3 Long-Term Safety Study of Migalastat

Phase 3

Terminated

Conditions: Fabry Disease

Interventions: Drug: migalastat hydrochloride

First Posted Date: 2011-10-24

Last Posted Date: 2018-10-02

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 85

Registration Number: NCT01458119

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