We found a match
Your institution may have access to this item. Find your institution then sign in to continue.
- Title
Cipaglucosidase Alfa: First Approval.
- Authors
Blair, Hannah A.
- Abstract
Cipaglucosidase alfa (Pombiliti™) is a recombinant human acid α-glucosidase (GAA) product being developed by Amicus Therapeutics along with the enzyme stabilizer miglustat as a two-component therapy for Pompe disease. Pompe disease is a rare, inherited lysosomal disease caused by a deficiency of the enzyme GAA, which leads to accumulation of glycogen in various tissues. On 27 March 2023, cipaglucosidase alfa was approved in the EU as a long-term enzyme replacement therapy (ERT) used in combination with miglustat for the treatment of adults with late-onset Pompe disease. This article summarizes the milestones in the development of cipaglucosidase alfa leading to this first approval.
- Subjects
DRUG approval; DRUG efficacy; NERVOUS system; INVESTIGATIONAL drugs; LYSOSOMAL storage diseases; PIPERIDINE; INBORN errors of carbohydrate metabolism; GLYCOSIDASES; DRUG development; ENZYME inhibitors; PHARMACODYNAMICS
- Publication
Drugs, 2023, Vol 83, Issue 8, p739
- ISSN
0012-6667
- Publication type
Article
- DOI
10.1007/s40265-023-01886-5