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Replagal (agalsidase alfa)
Agalsidase alfa is a recombinant human enzyme that replaces deficient α-galactosidase A, breaking down accumulated globotriaosylceramide in cells.
Agalsidase alfa is a recombinant human enzyme that replaces deficient α-galactosidase A, breaking down accumulated globotriaosylceramide in cells. Used for Fabry disease (α-galactosidase A deficiency).
At a glance
| Generic name | Replagal (agalsidase alfa) |
|---|---|
| Also known as | agalsidase alfa |
| Sponsor | Shire |
| Drug class | Enzyme replacement therapy |
| Target | α-galactosidase A (GLA) |
| Modality | Biologic |
| Therapeutic area | Rare genetic disease / Lysosomal storage disorder |
| Phase | FDA-approved |
Mechanism of action
Fabry disease is caused by deficiency of the lysosomal enzyme α-galactosidase A, leading to accumulation of the glycosphingolipid globotriaosylceramide (GL-3) in multiple tissues. Agalsidase alfa is a recombinant form of this enzyme that, when infused intravenously, enters cells and catalyzes the breakdown of GL-3, reducing pathological accumulation and halting or slowing disease progression. This enzyme replacement therapy addresses the underlying enzymatic defect in Fabry disease.
Approved indications
- Fabry disease (α-galactosidase A deficiency)
Common side effects
- Infusion reactions
- Fever
- Chills
- Headache
- Nausea
- Fatigue
Key clinical trials
- Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease (PHASE3)
- A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease (PHASE2, PHASE3)
- Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease (PHASE2, PHASE3)
- Galactol® Enzyme Supplement for Post-Prandial Abdominal Bloating in Irritable Bowel Syndrome (NA)
- A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease (PHASE3)
- Fabry Disease Registry & Pregnancy Sub-registry
- Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
- Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease (PHASE3)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |