COMPANY

Amicus Therapeutics

HQ: Princeton, NJ source · amicusrx.com ↗

Status: Acquired, now part of BioMarin Pharmaceutical Inc · How status works

Profile checked Oct 10, 2026

Keywords: Pompe Disease (Late-onset) Fabry Disease Pompe Disease Type 1 Gaucher Disease Gaucher Disease Glycogen Storage Disease Type II Infantile Onset Pompe Disease Infantile-Onset Late-onset Pompe Disease Epidermolysis Bullosa Glycogen Storage Disease Type II migalastat AT2221 ATB200 Afegostat Enzyme Replacement Therapy AT1001 AT2101

42Trials (company is sponsor)

Timeline

Feb 13, 2026 Trial start —: A Study of Patients With Fabry Disease (US Specific) source ↗
Jan 8, 2026 Trial start Phase 3: A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants source ↗
Mar 18, 2025 Trial start —: A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding source ↗
Feb 16, 2024 Trial start —: A Global Prospective Observational Registry of Patients With Pompe Disease source ↗
Jul 18, 2023 Trial start Phase 3: A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 source ↗
Mar 1, 2023 Trial start —: Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients source ↗
Oct 31, 2022 Trial start Phase 3: A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease source ↗
Mar 1, 2020 Trial start —: A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy. source ↗
Feb 13, 2020 Trial start Phase 3: ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD source ↗
Dec 18, 2019 Trial start Phase 3: A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD) source ↗
Oct 11, 2019 Trial start Phase 3: Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease source ↗
Dec 4, 2018 Trial start Phase 3: A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease source ↗
Sep 27, 2018 Trial start Phase 3: Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) source ↗
Dec 8, 2017 Trial start —: STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT source ↗
Apr 2, 2017 Trial start —: Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa source ↗
2016-04 Trial start Phase 1/Phase 2: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 source ↗
Mar 14, 2015 Trial start Phase 3: Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease source ↗
2014-03 Trial start 1: A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers source ↗
Oct 31, 2011 Trial start Phase 2: Drug-drug Interaction Study source ↗
Oct 14, 2011 Trial start 3: Open-Label Phase 3 Long-Term Safety Study of Migalastat source ↗
2011-10 Trial start Phase 1: Migalastat Food Effect Study source ↗
Sep 8, 2011 Trial start Phase 3: Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease source ↗
2011-09 Trial start I: 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 Health source ↗
2011-08 Trial start Phase 1: Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function source ↗
2011-08 Trial start Phase 1: A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014) source ↗

Showing 25 of 39 dated events.

Upcoming milestones

Dec 31, 2026 (ESTIMATED) · Phase 3 · A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease · NCT04020055
2027-03 (ESTIMATED) · Phase 3 · ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD · NCT03911505
2027-07 (ESTIMATED) · Phase 3 · A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 · NCT04808505
2027-12 (ESTIMATED) · Phase 3 · A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants · NCT06904261
2029-08 (ESTIMATED) · · A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding · NCT04252066
2032-06 (ESTIMATED) · · A Study of Patients With Fabry Disease (US Specific) · NCT06906367
Dec 20, 2034 (ESTIMATED) · · A Global Prospective Observational Registry of Patients With Pompe Disease · NCT06121011

Estimated primary completion per ClinicalTrials.gov. Dates move.

Clinical trials (lead sponsor) 42

Record	Title	Phase	Status	Start	Primary completion
NCT03911505	ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD	Phase 3	Active, not recruiting	Feb 13, 2020	2027-03 (estimated)
NCT06906367	A Study of Patients With Fabry Disease (US Specific)	Not listed	Recruiting	Feb 13, 2026	2032-06 (estimated)
NCT06904261	A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants	Phase 3	Recruiting	Jan 8, 2026	2027-12 (estimated)
NCT04020055	A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease	Phase 3	Active, not recruiting	Oct 31, 2022	Dec 31, 2026 (estimated)
NCT06121011	A Global Prospective Observational Registry of Patients With Pompe Disease	Not listed	Recruiting	Feb 16, 2024	Dec 20, 2034 (estimated)
NCT04138277	A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)	Phase 3	Completed	Dec 18, 2019	Dec 31, 2024 (actual)
NCT04281537	A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.	Not listed	Completed	Mar 1, 2020	May 18, 2022 (actual)
NCT04049760	Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease	Phase 3	Completed	Oct 11, 2019	Nov 29, 2024 (actual)
NCT04808505	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	Jul 18, 2023	2027-07 (estimated)
NCT02675465	First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221	Phase 1/Phase 2	Completed	2016-04	Aug 22, 2024 (actual)
NCT03865836	Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease	Not listed	Available	Not listed	Not listed
NCT00688597	Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease	Phase 2	Terminated	Dec 8, 2008	Dec 14, 2009 (actual)
NCT03729362	A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease	Phase 3	Completed	Dec 4, 2018	Dec 15, 2020 (actual)
NCT04327973	Expanded Access for ATB200/AT2221 for the Treatment of IOPD	Not listed	Available	Not listed	Not listed
NCT03347253	STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT	Not listed	Terminated	Dec 8, 2017	Nov 30, 2018 (actual)
NCT04252066	A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding	Not listed	Recruiting	Mar 18, 2025	2029-08 (estimated)
NCT01476163	Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease	Not listed	Available	Not listed	Not listed
NCT01380743	Drug-drug Interaction Study	Phase 2	Completed	Oct 31, 2011	Jan 4, 2013 (actual)
NCT04804566	Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients	Not listed	Completed	Mar 1, 2023	Nov 1, 2023 (actual)
NCT03500094	Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)	Phase 3	Completed	Sep 27, 2018	Feb 2, 2021 (actual)
NCT02194985	Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease	Phase 3	Completed	Mar 14, 2015	Oct 23, 2019 (actual)
NCT01196871	Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease	Phase 2	Completed	Feb 2, 2011	Oct 9, 2012 (actual)
NCT01218659	Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease	Phase 3	Completed	Sep 8, 2011	May 27, 2014 (actual)
NCT00283959	A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease	Phase 2	Completed	Jun 27, 2006	May 8, 2008 (actual)
NCT00214500	A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease	Phase 2	Completed	Jan 2, 2006	Jan 29, 2008 (actual)
NCT00925301	Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease	Phase 3	Completed	Oct 23, 2009	Jun 12, 2012 (actual)
NCT00526071	Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study	Phase 2	Terminated	Sep 17, 2007	Sep 8, 2012 (actual)
NCT00304512	A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease	Phase 2	Completed	Sep 7, 2006	May 9, 2008 (actual)
NCT01458119	Open-Label Phase 3 Long-Term Safety Study of Migalastat	3	Terminated	Oct 14, 2011	Feb 17, 2016 (actual)
NCT00433147	A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy	Phase 2	Completed	Mar 23, 2007	Feb 19, 2008 (actual)
NCT00283933	A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease	Phase 2	Completed	May 9, 2006	Mar 12, 2008 (actual)
NCT00446550	A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease	Phase 2	Completed	Jun 11, 2008	Aug 20, 2009 (actual)
NCT00813865	A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients	Phase 2	Completed	May 11, 2009	May 1, 2012 (actual)
NCT03158662	Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa	Not listed	Completed	Apr 2, 2017	Jun 30, 2017 (actual)
NCT01730469	Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function	Phase 1	Completed	2011-08	2012-04 (actual)
NCT02082327	A Phase 1 Study To Evaluate the Safety of Migalastat Hydrochloride Given Intravenously to Healthy Volunteers	1	Completed	2014-03	2014-06 (actual)
NCT01489995	Migalastat Food Effect Study	Phase 1	Completed	2011-10	2011-12 (actual)
NCT01730482	A Study to Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)	Phase 1	Completed	2011-08	2011-09 (actual)
NCT01853852	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	I	Completed	2011-09	2011-12 (actual)
NCT00351156	Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease	Not listed	Completed	2006-07	2007-03 (actual)
NCT00875160	A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101	Phase 1	Terminated	2009-04	2009-07 (actual)
NCT00515398	A Study to Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease	Not listed	Completed	2007-08	2008-01 (actual)