Data updated: Jun 28, 2026
Fabry Disease Clinical Trial Landscape
Fabry Disease is being studied across 61 clinical trials registered since 2008, with 14 programs currently active. The competitive pipeline includes 8 active Phase 3 trials, 5 active Phase 2 trials, and 0 active Phase 1 trials.
, Sanofi.
Trial activity
14 active / 61 total since 2008
Active by phase 8 Ph3 / 28 5 Ph2 / 18 0 Ph1 / 11 1 Ph4 / 4
Sponsor activity
Who is running trials now — green active, blue completed, red failed/terminated.
Sorted by active Active Done Failed
Amicus Therapeutics 2 11 1
Chiesi Farmaceutici S.p.A. 2 2 0
Sanofi 2 0 1
4D Molecular Therapeutics 2 0 0
Takeda 1 6 1
Idorsia Pharmaceuticals Ltd. 1 2 0
UniQure Biopharma B.V. 1 0 0
Guangzhou Jiayin Biotech Ltd 1 0 0
AceLink Therapeutics, Inc. 1 0 0
ISU Abxis Co., Ltd. 1 0 0
Genzyme, a Sanofi Company 0 5 1
Protalix 0 5 1
How the field has grown
New-trial starts peaked in 2022 (8 registered); 2025 saw 2. The right-hand chart shows median Phase 3 enrollment by start year — the number in parentheses is that year's Phase 3 trial count (19 in total), so single-trial years (and years with no Phase 3 starts) are obvious. Both are by trial start date; the current year is partial.
New trials started by year
2016 3
2017 3
2018 6
2019 3
2020 3
2021 3
2022 8
2023 3
2024 1
2025 2
2026 1
TheraRadar.com
Median Phase 3 enrollment by start year
2016 (1) 78
2017 (2) 26
2018 (5) 97
2019 (1) 16
2020 (0) 0
2021 (1) 17
2022 (5) 20
2023 (2) 20
2024 (0) 0
2025 (1) 22
2026 (1) 8
TheraRadar.com
Full trial pipeline
Every active and completed trial across Phase 1–4, with enrollment analytics. Sortable, filterable, exportable with Pro.
NCT06904261 RECRUITING
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Amicus Therapeutics n=8
NCT04020055 ACTIVE NOT RECRUITING
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Amicus Therapeutics n=14
NCT06328608 RECRUITING
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Chiesi Farmaceutici S.p.A. n=22
NCT05710692 RECRUITING
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Chiesi Farmaceutici S.p.A. n=16
NCT05280548 ACTIVE NOT RECRUITING
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
Sanofi n=104
NCT05206773 ACTIVE NOT RECRUITING
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease
Sanofi n=122
NCT03737214 ACTIVE NOT RECRUITING
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
Idorsia Pharmaceuticals Ltd. n=107
NCT06081062 RECRUITING
Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
ISU Abxis Co., Ltd. n=24
NCT03566017 COMPLETED
Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
Chiesi Farmaceutici S.p.A. n=97
NCT05843916 COMPLETED
Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease
Bio Sidus SA n=20
NCT04049760 COMPLETED
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Amicus Therapeutics n=16
NCT03614234 COMPLETED
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Chiesi Farmaceutici S.p.A. n=29
NCT04974749 COMPLETED
A Study of REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease
Takeda n=20
NCT03425539 COMPLETED
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Idorsia Pharmaceuticals Ltd. n=118
NCT02795676 COMPLETED
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
Protalix n=78
NCT03018730 COMPLETED
Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Protalix n=22
NCT03180840 COMPLETED
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
Protalix n=30
NCT03500094 COMPLETED
Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Amicus Therapeutics n=22
NCT00864851 COMPLETED
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
Shire n=44
NCT01298141 COMPLETED
A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
Shire n=171
NCT01124643 COMPLETED
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
Shire n=35
NCT02194985 COMPLETED
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
Amicus Therapeutics n=84
NCT01218659 COMPLETED
Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Amicus Therapeutics n=68
NCT00925301 COMPLETED
Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Amicus Therapeutics n=67
NCT01458119 TERMINATED
Open-Label Phase 3 Long-Term Safety Study of Migalastat
Amicus Therapeutics n=85
NCT02921620 WITHDRAWN
Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
Protalix
NCT00701415 COMPLETED
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
Genzyme, a Sanofi Company n=31
Data: ClinicalTrials.gov · Trials registered 2008 onwards · Industry sponsors only