Product Description
Sebelipase alfa (Kanuma®, Kanuma™), the first commercially available recombinant human lysosomal acid lipase (LAL), is approved in various countries worldwide, including those of the EU, the USA and Japan, as a long-term enzyme replacement therapy for patients diagnosed with LAL deficiency (LAL-D), an ultra-rare, autosomal recessive, progressive metabolic liver disease. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/27878737/)
Mechanisms of Action: cholesterol Hydrolyzer,triglyceride Hydrolyzer
Novel Mechanism: No
Modality: Coagulation Factor
Route of Administration: Intravenous
FDA Designation: None *
Approval Status: Approved
Approved Countries: Australia | Austria | Belgium | Brazil | Canada | Colombia | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Netherlands | Norway | Poland | Portugal | Romania | Russia | Saudi Arabia | Slovakia | Slovenia | Spain | Sweden | Switzerland | Turkey | United Arab Emirates | United Kingdom | United States
Approved Indications: None
Known Adverse Events: None
Company: AstraZeneca
Company Location: CAMBRIDGE X0 CB2 0AA
Company CEO: Pascal Soriot
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 3: Wolman Disease|Failure to Thrive
Phase 2: Cholesterol Ester Storage Disease|Wolman Disease|Other
Phase 1: Wolman Disease|Gaucher Disease|Glycogen Storage Disease Type II
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| IUERT | P1 |
Recruiting |
Glycogen Storage Disease Type II|Wolman Disease|Gaucher Disease |
2031-07-01 |
|
| ARISE (Acid Lipase Replacement Investigating Safety and Efficacy) | P3 |
Completed |
Unknown |
2018-12-11 |
|
| 2014-000533-22 | P2 |
Completed |
Other |
2018-10-30 |
|
| LAL-CL08 | P2 |
Terminated |
Wolman Disease |
2018-10-30 |