FDA APPROVES NEXVIAZYME, A NEWER TREATMENT FOR POMPE'S DISEASE

Edited by, JASPER VICTORIA

Pompe's disease is a debilitating glycogen storage disease, in which a complex sugar called glycogen builds up, disabling the heart and skeletal muscles, owing its etiology to mutations in the gene responsible for the enzyme acid α glucosidase, resulting in deficiency of the enzyme, acid α glucosidase, capable to cause muscle weakness, respiratory or heart failure leading to premature death.

The FDA approves an intravenous infusion, Nexviazyme (avalglucosidase alfa-ngpt) to treat late-onset Pompe's disease in both patients older and 1 year of age.

It is an enzyme replacement medication that could be used to aid in reduction of accumulating glycogen. Genzyme Corporation was granted the approval by the FDA for Nexviazyme to be used in Pompe's disease based on the study conducted with 100 patients administered with, Nexviazyme or a similar enzyme replacement medication, on which the individuals enrolled in the study showed improved lung function as in the other therapies available for Pompe's disease.

Diarrhea, nausea, vomiting, pruritus, urticaria, fatigue, dizziness, dyspnea, erythema, arthralgia, myalgia and paresthesia were the common side effects observed during the study and the notable serious reactions or adverse events were anaphylaxis, chills, respiratory distress and pyrexia. Cardiorespiratory failure is the risk associated with administering nexviazyme to patients with compromised cardiac or lung function or those susceptible to fluid volume overload.