Sebelipase Alfa (Kanuma) for treatment of lysosomal acid lipase deficiency
HAYES, Inc.
Record ID 32016000966
English
Authors' recommendations:
Description of Technology: This report focuses on the use of sebelipase alfa (Kanuma) for treatment of lysosomal acid lipase deficiency (LAL-D). Sebelipase alfa is a recombinant product with the same amino acid sequence as human lysosomal acid lipase (LAL). Sebelipase alfa replaces the missing LAL enzyme in LAL-D patients, helping to catalyze the lysosomal hydrolysis of cholesteryl esters and triglycerides to free cholesterol, glycerol, and free fatty acids. It is administered intravenously as a 2-hour infusion every other week, or weekly for patients with rapidly progressive LAL-D presenting within the first 6 months of life.
Patient Population: Sebelipase alfa is indicated for the treatment of LAL-D in adult and pediatric patients.
Details
Project Status:
Completed
Year Published:
2016
URL for published report:
The report may be purchased from: http://www.hayesinc.com/hayes/crd/?crd=44386
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
United States
MeSH Terms
- Lysosomal Storage Diseases
- Sterol Esterase
- Wolman Disease
- Enzyme Replacement Therapy
Contact
Organisation Name:
HAYES, Inc.
Contact Address:
157 S. Broad Street, Suite 200, Lansdale, PA 19446, USA. Tel: 215 855 0615; Fax: 215 855 5218
Contact Name:
saleinfo@hayesinc.com
Contact Email:
saleinfo@hayesinc.com
Copyright:
2014 Winifred S. Hayes, Inc
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.