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Label indication FABRAZYME ® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. FABRAZYME is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. ( 1 ) FDA label
Also known as Fabrazyme (agalsidase beta), AGALSIDASE BETA (GZ419828), agalsidase beta, r-hαGAL, r-haGAL, Agalsidase beta (recombinant form), Recombinant human α-galactosidase A (r-hαGAL)
Fabrazyme · 4 trials · 1 indication
Skin biopsies were taken at Baseline, Week 52, Week 156 and Week 260 or early withdrawal and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was scored for GL-3 accumulation on a severity score-scale of none, mild, moderate, severe (0-1-2-3). Scores are categorized as normal (score = 0) or abnormal (score = 1, 2 or 3). Data was summarized in terms of number of participants with none/trace, mild, moderate and severe biopsy scores.
Kidney biopsies were taken at Baseline, Week 24, and Week 96 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
Skin biopsies were taken at Baseline, Week 24 and Week 48 and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was evaluated by pathologists for the total number of vessels with GL-3 accumulation on an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe).
| Arm | Type | Description |
|---|---|---|
| Fabrazyme 0.5 mg/kg | EXPERIMENTAL | Fabrazyme 0.5 mg/kg was administered every 2 weeks (up to 131 infusion) up to 260 weeks, the total infusion time was not less than 45 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. |
| Fabrazyme 1.0 mg/kg | EXPERIMENTAL | Fabrazyme 1.0 mg/kg was administered every 4 weeks (up to 66 infusion) up to 260 weeks, the total infusion time was not less than 90 minutes. In case of significant progression of Fabry disease, the dose was increased to 1.0 mg/kg every 2 weeks. |
| Fabrazyme | EXPERIMENTAL | Open-label study. Patients received 1.0 mg/kg Fabrazyme every two weeks for approximately six months followed by 0.3 mg/kg Fabrazyme every two weeks for approximately 18 months. |
| Name | Type | Description |
|---|---|---|
| Agalsidase beta | BIOLOGICAL | Powder for concentrate for solution for infusion 1.0 mg/kg/4 weeks |
| Fabrazyme (agalsidase beta) | DRUG | - |
Inclusion Criteria: * The participant and/or participant's parent(s)/legal guardian(s) must provide written informed assent/consent prior to any protocol-related procedures being performed. * The participant must had a confirmed diagnosis of Fabry disease as documented by leukocyte α-Galactosidase ...
Fabrazyme is used to treat Fabry disease, a rare inherited lysosomal storage disorder. It is studied in both adult and pediatric patients with the condition, including males and mixed-sex populations across multiple countries. The clinical program covers patients as young as 7 years old.
Fabrazyme is developed by Sanofi, which trades under the ticker SNY. Sanofi is the sponsor of the clinical trials evaluating the therapy in Fabry disease, a rare disease indication within the company's portfolio.
Fabrazyme has been evaluated in Phase 3 clinical development for Fabry disease. Three trials are registered in total, all of which are completed, including one Phase 3 study and two Phase 2 studies. No trials are currently active.
Fabrazyme has been studied in three completed trials: NCT00074971, a Phase 3 study in adults across the United States, France, Netherlands, Puerto Rico, and the United Kingdom; NCT00196716, a Phase 2 study in Czechia, Estonia, Poland, and Slovakia; and NCT00074958, a Phase 2 pediatric study.
Yes, Fabrazyme is also known as agalsidase beta. The two names refer to the same therapy developed by Sanofi for Fabry disease. Readers searching for agalsidase beta are looking at the same clinical program, which includes three completed trials in Fabry disease.