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

A Study of Patients With Fabry Disease (US Specific)

Not yet recruiting

Conditions: Fabry Disease

Interventions: Drug: migalastat HCl
Drug: ERT

First Posted Date: 2025-04-02

Last Posted Date: 2025-04-04

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 450

Registration Number: NCT06906367

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Phase 3

Recruiting

Conditions: Fabry Disease

Interventions: Drug: Migalastat HCl 20 mg

First Posted Date: 2025-04-01

Last Posted Date: 2025-07-03

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 8

Registration Number: NCT06904261

Locations: 🇺🇸
Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax, Virginia, United States

A Global Prospective Observational Registry of Patients With Pompe Disease

Recruiting

Conditions: Pompe Disease

Interventions: Other: Untreated
Biological: Alglucosidase alfa or Avalglucosidase alfa
Biological: Cipaglucosidase alfa
Drug: Miglustat

First Posted Date: 2023-11-07

Last Posted Date: 2025-07-03

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 500

Registration Number: NCT06121011

Locations: 🇺🇸
University of Arkansas Medical Science, Little Rock, Arkansas, United States
🇺🇸
University of California Irvine, Irvine, California, United States
🇺🇸
Wolfson Children's Hospital, Jacksonville, Florida, United States

and more 22 locations

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Phase 3

Recruiting

Conditions: Glycogen Storage Disease Type II Infantile Onset

Interventions: Biological: Cipaglucosidase alfa
Drug: Miglustat

First Posted Date: 2021-03-22

Last Posted Date: 2025-05-30

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 36

Registration Number: NCT04808505

Locations: 🇺🇸
University of Florida Clinical Research Center, Gainesville, Florida, United States
🇺🇸
The Emory Clinic, Atlanta, Georgia, United States
🇺🇸
Duke University Early Phase Research Unit, Durham, North Carolina, United States

and more 13 locations

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Completed

Conditions: Fabry Disease

Interventions: Other: This is a non-interventional study

First Posted Date: 2021-03-18

Last Posted Date: 2024-04-03

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 44

Registration Number: NCT04804566

Locations: 🇺🇸
Engage Health, Eagan, Minnesota, United States

Natural History Study of Batten Disease

Terminated

Conditions: Neuronal Ceroid Lipofuscinosis CLN6
Neuronal Ceroid Lipofuscinosis CLN3
Batten Disease

First Posted Date: 2020-11-25

Last Posted Date: 2022-04-11

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 10

Registration Number: NCT04644549

Locations: 🇺🇸
University of Rochester Medical Center, Rochester, New York, United States

Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Conditions: Pompe Disease Infantile-Onset

First Posted Date: 2020-03-31

Last Posted Date: 2024-08-16

Lead Sponsor: Amicus Therapeutics

Registration Number: NCT04327973

Locations: 🇺🇸
UCSF Benioff Children's Hospital Oakland, Oakland, California, United States
🇺🇸
UF Health Shands Hospital, Gainesville, Florida, United States
🇮🇹
University Hospital of Padova, Padova, Italy

and more 1 locations

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

Completed

Conditions: Fabry Disease

Interventions: Drug: Enzyme Replacement Therapy (ERT)

First Posted Date: 2020-02-24

Last Posted Date: 2024-11-04

Lead Sponsor: Amicus Therapeutics

Target Recruit Count: 82

Registration Number: NCT04281537

Locations: 🇺🇸
Amicus Therapeutics, Inc., Philadelphia, Pennsylvania, United States
🇧🇷
Instituto de Genética e Erros Inatos do Metabolismo (IGEIM), São Paulo, Brazil
🇯🇵
Keio University Hospital, Tokyo, Japan

and more 6 locations