Product Description
Agalsidase beta (Fabrazyme) is a recombinant human alpha-galactosidase A enzyme approved for intravenous use in the treatment of Fabry disease. Fabry disease is a progressive, multisystemic, potentially life threatening disorder caused by a deficiency of alpha-galactosidase A. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/17335299/)
Mechanisms of Action: Glycosphingolipid Agonist
Novel Mechanism: No
Modality: Coagulation Factor
Route of Administration: Intravenous
FDA Designation: None *
Approval Status: Approved
Approved Countries: Algeria | Argentina | Australia | Austria | Belgium | Bosnia | Brazil | Canada | Chile | Colombia | Croatia | Cyprus | Czech | Denmark | Dominican Republic | Ecuador | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hong Kong | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lebanon | Lithuania | Luxembourg | Malaysia | Netherlands | New Zealand | Norway | Peru | Philippines | Poland | Portugal | Romania | Russia | Saudi Arabia | Serbia | Singapore | Slovakia | Slovenia | South Africa | Spain | Sweden | Switzerland | Taiwan | Thailand | Tunisia | Turkey | Ukraine | United Arab Emirates | United Kingdom | United States | Uruguay | Venezuela
Approved Indications: Fabry Disease
Known Adverse Events: Dizziness | Headache | Paresthesia | Respiratory Tract Infections | Chills | Edema
Company: Sanofi
Company Location: PARIS I0 75008
Company CEO: Paul Hudson
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Argentina, China, Korea, Philippines
Active Clinical Trial Count: 4
Highest Development Phases
Phase 3: Fabry Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| ISU303-003 | P3 |
Recruiting |
Fabry Disease |
2025-06-01 |
|
| SMILE | P3 |
Recruiting |
Fabry Disease |
2023-12-01 |
95% |
| AGA Biosidus | P1 |
Completed |
Fabry Disease |
2022-03-06 |
|
| CTR20220800 | P3 |
Not yet recruiting |
Fabry Disease |
None |