Regulatory news: Cipaglucosidase alfa-atga (Pombiliti) coadministered with Miglustat (Opfolda) for adults with late-onset Pompe disease

J Inherit Metab Dis. 2024 Jul;47(4):578-581. doi: 10.1002/jimd.12744. Epub 2024 May 20.

Authors

Daniela V Luquetti¹, Linda J B Jeng¹, Kathleen M Donohue¹, Janet W Maynard¹; Review Team^{2

3

4}

Collaborators

Affiliations

¹ Office of New Drugs, Center for Drug Evaluation and Research, Silver Spring, Maryland, USA.
² Office of Biostatistics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
³ Office of Clinical Pharmacology, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
⁴ Office of Pharmaceutical Quality, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.

PMID: 38768612
DOI: 10.1002/jimd.12744

No abstract available

Publication types

Editorial

MeSH terms

1-Deoxynojirimycin* / administration & dosage
1-Deoxynojirimycin* / adverse effects
1-Deoxynojirimycin* / analogs & derivatives
1-Deoxynojirimycin* / therapeutic use
Adult
Age of Onset
Drug Approval
Drug Therapy, Combination
Enzyme Replacement Therapy
Glycogen Storage Disease Type II* / drug therapy
Humans
alpha-Glucosidases*

Substances

alpha-Glucosidases
1-Deoxynojirimycin
miglustat
GAA protein, human