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Fabrazyme (agalsidase beta)
Fabrazyme (agalsidase beta) is a marketed enzyme replacement therapy developed by Genzyme (Sanofi) for Fabry disease in patients aged 2 years and older. The drug provides exogenous α-galactosidase A to reduce pathogenic GL-3 accumulation in lysosomes. With 38 clinical trials and 293 publications, Fabrazyme represents a foundational treatment for this rare lysosomal storage disorder. The drug demonstrates clinical differentiation through its established safety and efficacy profile in both pediatric and adult populations. As a pioneering enzyme replacement therapy, Fabrazyme maintains significant commercial importance in the rare disease market, though newer alternatives have emerged.
At a glance
| Generic name | Fabrazyme (agalsidase beta) |
|---|---|
| Also known as | r-hαGAL, Fabrazyme |
| Sponsor | Genzyme, a Sanofi Company |
| Drug class | Enzyme replacement therapy (ERT); recombinant enzyme |
| Target | α-galactosidase A (GLA gene product); substrate: globotriaosylceramide (GL-3) |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
Approved indications
- Treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease
Boxed warnings
- WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate FABRAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitiv
Common side effects
Drug interactions
- Anticoagulants
- Immunosuppressants
- Anti-inflammatory drugs
Key clinical trials
- 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
- Switch Over Study of Biosimilar Agalsidase Beta for Fabry Disease (PHASE3)
- A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
- Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
- A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (PHASE4)
- PK/PD Study of 2 Agalsidase Formulations in Single Dose of 1 mg/kg Administered to Healthy Volunteers as IV Infusion (PHASE1)
- China Post-marketing Surveillance (PMS) Study of Fabrazyme® (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Fabrazyme (agalsidase beta) CI brief — competitive landscape report
- Fabrazyme (agalsidase beta) updates RSS · CI watch RSS
- Genzyme, a Sanofi Company portfolio CI