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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 Agalsidase beta (GZ419828), Fabrazyme, Recombinant human α-galactosidase A (r-hαGAL)
Agalsidase beta · 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 ...
Agalsidase beta is an investigational drug being studied for the treatment of Fabry Disease, a rare genetic disorder. It is currently in Phase 3 clinical development. The drug is being evaluated in patients with this condition to assess its safety and efficacy.
Agalsidase beta is being developed by Sanofi, a global biopharmaceutical company. Sanofi is listed on the stock exchange under the ticker symbol SNY. The company is conducting clinical trials to evaluate the drug's potential in treating Fabry Disease.
Agalsidase beta is currently in Phase 3 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. The drug is being studied for the treatment of Fabry Disease in clinical trials.
Agalsidase beta has one completed Phase 3 clinical trial with the identifier NCT00701415. This trial studied two dosing regimens of the drug in treatment-naive, male pediatric patients with Fabry Disease. The trial enrolled 31 participants across multiple countries.
Yes, Agalsidase beta is also known as GZ419828. This alternative name may be used in some clinical or research contexts. The drug is being developed by Sanofi for the treatment of Fabry Disease.