sebelipase alfa

Chemical data

Drug data · ChEMBL

Max clinical phase

Approved

Molecule type

Enzyme

First approval

2015

Admin. routes

parenteral

Indications

Wolman disease, cholesteryl ester storage disease, lysosomal acid lipase deficiency, inborn errors of metabolism

via ChEMBL · EBI

💊Medication

Brand names

Kanuma

Drug class

Enzymes

Active ingredients

sebelipase alfa

Dosage form

Injection

• PHASE2COMPLETED
  Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase DeficiencyAlexion Pharmaceuticals, Inc. · NCT01488097
• NO_LONGER_AVAILABLE
  An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase DeficiencyAlexion Pharmaceuticals, Inc. · NCT02376751
• PHASE2/PHASE3COMPLETED
  Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyAlexion Pharmaceuticals, Inc. · NCT01371825
• PHASE3COMPLETED
  Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase DeficiencyAlexion Pharmaceuticals, Inc. · NCT01757184
• PHASE1/PHASE2COMPLETED
  Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase DeficiencyAlexion Pharmaceuticals, Inc. · NCT01307098
• PHASE2TERMINATED
  Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase DeficiencyAlexion Pharmaceuticals, Inc. · NCT02193867

via ClinicalTrials.gov

Wikidata facts

Instance of

enzyme

Subclass of

medication

Mass

43003.4822

via Wikidata · CC0