Recombinant Human Acid .alpha.-glucosidase
Recombinant Human Acid .alpha.-glucosidase Uses, Dosage, Side Effects, Food Interaction and all others data.
Recombinant Human Acid .alpha.-glucosidase, or NeoGAA, is an experiemental enzyme replacement therapy designed for Pompe disease, which is a rare inherited neuromuscular disorder caused by mutations in the GAA gene, which encodes the lysosomal hydrolase acid alpha-glucosidase (GAA). GAA is an essential enzyme for lysosomal glycogen catabolism; thus, GAA deficiency leads to accumulation of glycogen within lysosomes and progressive disruption of cellular function, especially in smooth, cardiac, and skeletal muscle cells. Pompe disease is characterized by progressive muscle weakness and loss of motor function, including respiratory muscle weakness, which is the leading cause of death in late-onset Pompe disease. Recombinant Human Acid .alpha.-glucosidase is a recombinant form of GAA to restore the deficient enzyme levels. First developed by Sanofi Genzyme, avalglucosidase alfa is a chemically modified version of alglucosidase alfa, where synthetic bis-phosphorylated oligosaccharides were attached to the structure to improve cellular uptake of the drug and better muscle targeting.
Recombinant Human Acid .alpha.-glucosidase is under investigation in clinical trial NCT02782741 (Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease).
| Trade Name | Recombinant Human Acid .alpha.-glucosidase |
| Generic | Avalglucosidase alfa |
| Avalglucosidase alfa Other Names | Avalglucosidase alfa, Neo-recombinant human acid alpha-glucosidase, NeoGAA, Recombinant human acid .alpha.-glucosidase |
| Type | |
| Groups | Investigational |
| Therapeutic Class | |
| Manufacturer | |
| Available Country | |
| Last Updated: | September 19, 2023 at 7:00 am |
Uses
Recombinant Human Acid .alpha.-glucosidase is a long-term acid alpha-glucosidase enzyme replacement therapy being investigated for Pompe disease.
Innovators Monograph
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