Organization (Industry)
Amicus Therapeutics (United States)
ClinicalTrials.gov lists Amicus Therapeutics (United States) as lead sponsor of 42 trials. It has 19 active development programs and 9 approved assets.
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Trials started per year with this organization as sponsor or collaborator, 1990 onward. The series also counts trials where the organization is a collaborator; the lead-sponsor trial count does not.
2026 is partial: 2 registered with a 2026 start date. The dashed outline is the pace from the 2 registered with a start date through September, planned starts included, not adjusted; not a forecast. No forecast is made for this organization: it averages 1.6 trial starts a year over the five years to September, under the 5 a year the method needs.
This page covers Amicus Therapeutics (United States): 42 lead-sponsored trials and 2 legal-entity registrations.
Disease areas (14)
Shown in this order: most trials first.
| Disease area | Trials |
|---|---|
| Cerebral Small Vessel Diseases | 28 |
| Fabry Disease | 28 |
| Glycogen Storage Disease Type II | 11 |
| Epidermolysis Bullosa | 5 |
| Gaucher Disease | 5 |
Showing 5 of 14 disease areas.
Trials by start year as sponsor or collaborator (17)
Shown in this order: most recent first. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Show the table of trials by start year as sponsor or collaborator
| Year | Trials started |
|---|---|
| 2026 (partial year) | 2 |
| 2025 | 1 |
| 2024 | 1 |
| 2023 | 3 |
| 2022 | 1 |
| 2020 | 2 |
| 2019 | 4 |
| 2018 | 3 |
| 2017 | 4 |
| 2016 | 2 |
Showing 10 of 17 years.
Recruitment duration, a model prediction: 4 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
Active development programs (19)
Shown in this order: max phase, then active trials.
| Drug | Disease area | Max phase | Trials |
|---|---|---|---|
| Migalastat | Fabry Disease | Phase 3 | 11 |
| Migalastat | Cerebral Small Vessel Diseases | Phase 3 | 11 |
| Miglustat | Glycogen Storage Disease Type II | Phase 3 | 3 |
| Cipaglucosidase Alfa | Glycogen Storage Disease Type II | Phase 3 | 3 |
| Migalastat HCl 150 mg | Cerebral Small Vessel Diseases | Phase 3 | 5 |
Showing 5 of 19 active programs.
Approved assets (9)
Shown in this order: max phase first.
| Drug | Trials |
|---|---|
| Migalastat | 11 |
| ATB200 | 4 |
| Cipaglucosidase Alfa | 4 |
| Miglustat | 4 |
| ALGLUCOSIDASE ALFA | 2 |
Showing 5 of 9 approved assets.
Sponsor delivery record (1)
Enrollment hit target: the share of this sponsor's lead-sponsored trials whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 40% hit target (n=10) | 22% on time (n=9) |
Journals published in (41)
Shown in this order: most published in first.
| Journal | Publications |
|---|---|
| Genetics in medicine : official journal of the American College of Medical Genetics | 5 |
| Muscle & nerve | 5 |
| Orphanet journal of rare diseases | 5 |
| Journal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance | 4 |
| Circulation research | 3 |
Showing 5 of 41 journals.
US granted patents (1)
Patents are not classified by disease area in the source, so this is the organization's overall portfolio, not a split by disease.
| US patents granted | Granted in the last 5 years | Most recent grant |
|---|---|---|
| 157 | 57 | 2025-09-23 |
Financing and grant events (2)
Shown in this order: most recent first.
| Date | Round type | Amount (USD) | Source |
|---|---|---|---|
| 2023-10-05 | Private placement | 29,999,985 | Form d |
| 2021-09-29 | Private placement | 199,916,500 | Form d |
FDA inspection record (1)
| Latest classification | Inspections |
|---|---|
| NAI | 1 |
Organization relationships (10)
Shown in this order: most evidence first.
| Related organization | Relationship | Evidence |
|---|---|---|
| Fédération française de cardiologie | Co sponsors trial with | 1 |
| Ospedale San Donato | Co sponsors trial with | 1 |
| General University Hospital in Prague | Co sponsors trial with | 1 |
| Caroline Michaela Kistorp | Co sponsors trial with | 1 |
| Copenhagen University Hospital, Rigshospitalet | Co sponsors trial with | 1 |
Showing 5 of 10 relationships.
Latest dated events (210)
Shown in this order: newest first.
| Date | Event | Source |
|---|---|---|
| 2026-02-13 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2026-01-08 | Trial started | Clinical trials (ClinicalTrials.gov) (ClinicalTrials.gov, U.S. National Library of Medicine; AACT database, Clinical Trials Transformation Initiative (CTTI)) |
| 2025-09-23 | Patent granted | Granted US patents (PatentsView / USPTO) |
| 2025-09-16 | Patent granted | Granted US patents (PatentsView / USPTO) |
| 2025-09-16 | Patent granted | Granted US patents (PatentsView / USPTO) |
Showing 5 of 210 events.
Biomarker capability evidence (75)
Shown in this order: tier desc, then gene symbol.
| Gene | Role | Evidence |
|---|---|---|
| ACE | Trial sponsor | Named in trial inclusion criteria |
| ACE | Trial sponsor | Named in trial inclusion criteria |
| EPHA3 | Trial sponsor | Named in trial inclusion criteria |
| EPHA3 | Trial sponsor | Named in trial inclusion criteria |
| GAA | Trial sponsor | Named in trial inclusion criteria |
Showing 5 of 75 evidence rows.
Not on this page
Checked, with nothing recorded for this organization: companies grouped under this page, related companies not grouped here, sec-reported annual financials, fda warning letters, fda untitled letters, fda recalls, fda import refusals, manufacturing and distribution sites, climate targets, as filed, workforce and board diversity, as filed, clia clinical laboratory certificates, and biomarkers this organization publishes on.