Avalglucosidase alfa-ngpt

Avalglucosidase alfa-ngpt' is a recombinant human GAA (acid alpha-glucosidase) enzyme approved for the treatment of Pompe disease, a rare, inherited lysosomal storage disorder characterized by the accumulation of glycogen in the lysosomes of cells. Marketed under the brand name Nexviazyme, avalglucosidase alfa-ngpt is designed to enhance the clearance of glycogen from lysosomes, thereby improving muscle function and reducing the symptoms associated with the disease.

Medical Use

Avalglucosidase alfa-ngpt is indicated for the treatment of patients with late-onset Pompe disease. The therapy aims to improve respiratory function and increase mobility in affected individuals by replacing the deficient GAA enzyme, facilitating the breakdown and clearance of glycogen in lysosomes.

Mechanism of Action

Avalglucosidase alfa-ngpt functions by substituting the deficient or absent GAA enzyme in patients with Pompe disease. This enzyme is crucial for the breakdown of glycogen into glucose within lysosomes. By providing a recombinant form of GAA, avalglucosidase alfa-ngpt aids in the reduction of glycogen accumulation in cells, particularly in muscle tissues, thereby alleviating the symptoms of muscle weakness and respiratory difficulties.

Clinical Trials

Clinical trials of avalglucosidase alfa-ngpt have demonstrated its efficacy in improving motor function and stabilizing or improving respiratory function in patients with Pompe disease. These studies have played a critical role in the approval and subsequent recommendation of avalglucosidase alfa-ngpt as a treatment option for this condition.

Side Effects

The most common side effects associated with avalglucosidase alfa-ngpt include headache, fatigue, diarrhea, nausea, dizziness, and hypersensitivity reactions. Infusion-related reactions are also reported, which are typically manageable with premedication or adjustment of the infusion rate.

Approval and Regulation

Avalglucosidase alfa-ngpt received approval from the Food and Drug Administration (FDA) in August 2021 for the treatment of Pompe disease. This approval was based on the positive outcomes observed in clinical trials, highlighting the drug's potential to significantly improve the quality of life for patients with this condition.

See Also

• Enzyme replacement therapy
• Lysosomal storage disorders
• Glycogen storage disease type II