agalsidase alfa
Trade name: Replagal
α-Galactosidase is a glycoside hydrolase enzyme that catalyses the following reaction:Hydrolysis of terminal, non-reducing α-D-galactose residues in α-D-galactosides, including galactose oligosaccharides, galactomannans and galactolipids — Wikipedia
Clinical trial activity
23 trials · 45 clinical orgs · 0 marketing orgs
Earliest trial started Sep 1, 2001 (NCT01997489)
Timeline
Indications
Approved for
Studied for
Mechanism of action
- Alpha-galactosidase AACTIVATOR
GLA activator
Agalsidase alfa catalyses the hydrolysis of globotriaosylceramide (Gb3), cleaving a terminal galactose residue from the molecule. It counteracts the deficient activity of the lysosomal enzyme alpha-galactosidase A in the Fabry disease. Treatment with the enzyme has been shown to reduce accumulation of Gb3 in many cell types including endothelial and parenchymal cells.
- GlobotriosylceramideHYDROLYTIC ENZYME
Globotriosylceramide
- Alpha-galactosidase ASupplementation
GLA supplementation
Agalsidase alfa catalyses the hydrolysis of globotriaosylceramide (Gb3), cleaving a terminal galactose residue from the molecule. It counteracts the deficient activity of the lysosomal enzyme alpha-galactosidase A in the Fabry disease. Treatment with the enzyme has been shown to reduce accumulation of Gb3 in many cell types including endothelial and parenchymal cells.
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Multi-specific
- No
Oral
No
Parenteral
No
Topical
No
Sources
- WikipediaΑ-Galactosidase ↗
- ChEMBLCHEMBL2108214 ↗
- WikipediaEnzyme replacement therapy ↗
- WikipediaAlpha-galactosidase ↗
Also known as
- agalsidase
- agalsidase alfa
- agalsidase alfa
- agalsidase alfa
- agalsidase alfa
- agalsidase alfa
- agalsidase alfa
- agalsidase alfa
- agalsidase alfa (genetical recombination)
- agalsidase alpha
- drx005b
- enzyme replacement therapy
- replagal
- replagal
- replagal
- replagal
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT05280548 | Phase 3 | May 3, 2022 | Genzyme, Sanofi |
| NCT05067868 | Phase 4 | Jan 31, 2022 | Shire, Takeda |
| NCT04974749 | Phase 3 | Dec 31, 2021 | Takeda |
| NCT04840667 | Phase 3 | Nov 8, 2021 | Shire |
| NCT04143958 | Phase 4 | Dec 2, 2019 | Sanofi |
| NCT02956954 | N/A | Dec 1, 2016 | Rouen Normandy University |
| NCT02795676 | Phase 3 | Jun 1, 2016 | Protalix |
| NCT02930655 | Phase 1 | Feb 1, 2015 | Actelion |
| NCT01298141 | Phase 3 | May 1, 2011 | Shire |
| NCT01363492 | Phase 2 | May 1, 2011 | Baylor University, Duke University, Emory University, O & O Alpan LLC, Shire, University of Utah |
| NCT01304277 | Phase 2 | Mar 1, 2011 | Shire |
| NCT01218659 | Phase 3 | Dec 1, 2010 | Amicus Therapeutics |
| NCT01124643 | Phase 3 | Apr 1, 2010 | Charles University, General Hospital Slovenj Gradec, Melbourne Health, O & O Alpan LLC, Salford Royal Foundation NHS Trust, Shire, Szpital Uniwersytecki w Krakowie, University of Iowa, University of Turku |
| NCT01031173 | N/A | Dec 8, 2009 | Baylor University, Carilion New River Valley Medical Center, Central Coast Nephrology, Denver Nephrologists, P.C., Emory University, Infusion Associates, Kaiser Permanente, Mission Health, North Carolina, New York University, O & O Alpan LLC, Oregon Health and Science University, Shire, St. Joseph's Regional Medical Center, Wisconsin, Tidewater Kidney Specialists, University of Arizona, University of California, Davis, University of California, San Diego, University of California, San Francisco, University of Iowa, University of Massachusetts, University of Missouri, University Research Foundation for Lysosomal Storage Disorders |
| NCT00864851 | Phase 3 | Dec 1, 2008 | Charles University, General Hospital Slovenj Gradec, Melbourne Health, New York University, O & O Alpan LLC, Salford Royal Foundation NHS Trust, Shire, Szpital Uniwersytecki w Krakowie, University of Arizona, University of Iowa, University of Turku |
| NCT00097890 | Phase 4 | Nov 1, 2004 | National Institute of Neurological Disorders and Stroke (NINDS) |
| NCT00084084 | Phase 2 | Jun 1, 2004 | Baylor University, Christus St. Patrick Hospital, National Institutes of Health (NIH), New York University, Shire, Tuscon Access Center of Arizona Kidney Disease Hypertension Center, University of Arizona, University of Tennessee, University of Toronto, Washington University in St. Louis |
| NCT00075244 | Phase 2 | Jan 1, 2004 | National Institute of Neurological Disorders and Stroke (NINDS) |
| NCT00071877 | Phase 2 | Oct 1, 2003 | National Institute of Neurological Disorders and Stroke (NINDS) |
| NCT00357786 | Phase 1 | Oct 1, 2003 | National Institute of Neurological Disorders and Stroke (NINDS) |
Organizations
Research & Development (45)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Shire | For profit | 9 | 9 | 4 | 2004 |
| National Institute of Neurological Disorders and Stroke (NINDS) | Government | 6 | 5 | 3 | 2002 |
| Baylor University | Academic/Hospital | 4 | 1 | 2 | 2003 |
| O & O Alpan LLC | Academic/Hospital | 4 | 0 | 3 | 2008 |
| New York University | Academic/Hospital | 3 | 0 | 3 | 2004 |
| University of Arizona | Academic/Hospital | 3 | 0 | 3 | 2004 |
| University of Iowa | Academic/Hospital | 3 | 0 | 2 | 2008 |
| Charles University | Academic/Hospital | 2 | 0 | 1 | 2008 |
| Emory University | Academic/Hospital | 2 | 0 | 2 | 2009 |
| General Hospital Slovenj Gradec | Academic/Hospital | 2 | 0 | 1 | 2008 |
| Melbourne Health | Academic/Hospital | 2 | 0 | 1 | 2008 |
| Salford Royal Foundation NHS Trust | Academic/Hospital | 2 | 0 | 1 | 2008 |
| Sanofi | For profit | 2 | 1 | 2 | 2019 |
| Szpital Uniwersytecki w Krakowie | Academic/Hospital | 2 | 0 | 1 | 2008 |
| Takeda | For profit | 2 | 1 | 2 | 2021 |
| University of Turku | Academic/Hospital | 2 | 0 | 1 | 2008 |
| Actelion | For profit | 1 | 1 | 1 | 2015 |
| Amicus Therapeutics | For profit | 1 | 1 | 1 | 2010 |
| Carilion New River Valley Medical Center | Academic/Hospital | 1 | 0 | 1 | 2009 |
| Central Coast Nephrology | Academic/Hospital | 1 | 0 | 1 | 2009 |