Fabry´s disease

9 trials 3 recruiting MedDRA PT
9 Registered trials
3 Recruiting
6 Unique sponsors
8 Unique IMPs
8 Commercial trials
Drug classes (ATC)
— ATC code not specified 8
A Gastroenterology (ATC A) 1
Mechanism of action (INN-stem analysis)
Unknown / small molecule 8 substances
Phase III: 6 Phase II/III: 1 Phase II: 1 Phase I (First in Human): 1

Filter these 9 trials by country, sponsor, date range and more.

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Show: 9 trials

Phase III

6 trials
  • Amicus Therapeutics Inc. Commercial
    MIGALASTAT HYDROCHLORIDE
    2025-521244-38-00 3 countries n=3 Orphan
    Pending
  • Chiesi Farmaceutici S.p.A. Commercial
    PEGUNIGALSIDASE ALFA
    2024-516735-27-00 5 countries 2019 n=10 Orphan
    Ended
  • Idorsia Pharmaceuticals Ltd. Commercial
    LUCERASTAT
    2024-513884-20-00 7 countries 2019 n=45 Orphan
    Ended
  • Sanofi-Aventis Recherche & Developpement Commercial
    AGALSIDASE ALFA; AGALSIDASE BETA; MIGALASTAT; VENGLUSTAT
    2023-509715-91-00 11 countries 2022 n=57 Orphan
    Recruiting
  • Sanofi-Aventis Recherche & Developpement Commercial
    VENGLUSTAT
    2024-511990-31-00 10 countries 2022 n=48 Orphan
    Ended
  • Medical University Of Lodz Academic
    IMP not specified
    2025-521408-24-00 1 country 2026 n=40
    Recruiting

Phase II/III

1 trial
  • Chiesi Farmaceutici S.p.A. Commercial
    PEGUNIGALSIDASE ALFA
    2022-503128-29-00 4 countries 2024 n=7
    Recruiting

Phase II

1 trial
  • Sangamo Therapeutics Inc. Commercial
    ISARALGAGENE CIVAPARVOVEC
    2024-512700-18-00 1 country 2025 n=1 Orphan
    Ended

Phase I (First in Human)

1 trial
  • Sangamo Therapeutics Inc. Commercial
    ISARALGAGENE CIVAPARVOVEC
    2024-512695-34-00 2 countries 2022 n=8 Orphan
    Ended
Sponsor activity
SponsorTypeTrialsPhasesRecruiting
Chiesi Farmaceutici S.p.A. Commercial 2 Phase II/III, Phase III 1
Sangamo Therapeutics Inc. Commercial 2 Phase II, Phase I (First in Human) 0
Sanofi-Aventis Recherche & Developpement Commercial 2 Phase III 1
Amicus Therapeutics Inc. Commercial 1 Phase III 0
Idorsia Pharmaceuticals Ltd. Commercial 1 Phase III 0
Medical University Of Lodz Academic 1 Phase III 1
New trials by year
2019
2
2022
3
2024
1
2025
1
2026
1
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