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# Genzyme BLA Approved for Pompe Disease
- URL: https://www.fdaweb.com/genzyme-bla-approved-for-pompe-disease/
- Published: 2021-08-06T12:00:00.000Z
- Updated: 2026-09-14T17:09:39.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5149886

FDA has approved a Genzyme BLA for Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients one year of age and older with late-onset Pompe disease. The intravenous enzyme replacement therapy helps reduce glycogen accumulation, according to an FDA [news release](https://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pompe-disease?ref=fdaweb.com). In Pompe patients, glycogen accumulation in skeletal and heart muscles can cause muscle weakness and premature death from respiratory or heart failure.  

Approval was based on data from a 100-patient study that demonstrated the therapy’s ability to provide patients with improvements in respiratory function and walking distance. The most common side effects included headache, fatigue, diarrhea, nausea, joint pain, dizziness, muscle pain, itching, vomiting, difficulty breathing, skin redness and skin welts, according to FDA.