avalglucosidase alfa
Orphan Drug Cold Chain RequiredFDA Approved (2021), EMA Approved (2021)
Description
Avalglucosidase alfa is a next-generation enzyme replacement therapy for Pompe disease (acid alpha-glucosidase deficiency). It is designed with enhanced uptake into target tissues via mannose-6-phosphate receptors. Clinical trials demonstrated superior respiratory and motor function improvements compared to the previous standard alglucosidase alfa, particularly in late-onset Pompe disease patients.
Indications & Therapeutic Use
Pompe disease (acid alpha-glucosidase deficiency) in adults and pediatric patients
Global Availability (7 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
avalglucosidase alfa
| Generic Name | avalglucosidase alfa |
| Brands | 1 brand available |
| Active Ingredient | avalglucosidase alfa-ngpt |
| Drug Class | Pompe disease (acid alpha-glucosidase deficiency) in adults and pediatric patients |
| Manufacturer | Sanofi Genzyme |
| Dosage Forms | Intravenous infusion, 100mg lyophilized powder |
| Medical Code | A16AB13 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 21 days |
| Reg. Status | FDA Approved (2021), EMA Approved (2021) |
| Clinical Trial | NCT02782741 |
| Countries | 7 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations8 Validated Nodes