alglucosidase alfa
Orphan Drug Cold Chain RequiredFDA Approved, EMA Approved
Description
Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme replacement therapy. It is indicated for the treatment of Pompe disease (glycogen storage disease type II), including infantile-onset forms that can present with severe cardiomyopathy. The enzyme catalyzes the hydrolysis of glycogen to glucose in lysosomes.
Indications & Therapeutic Use
Pompe disease, glycogen storage disease type II, infantile-onset Pompe disease with cardiomyopathy
Linked Diseases:
Global Availability (7 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
alglucosidase alfa
| Generic Name | alglucosidase alfa |
| Brands | 1 brand available |
| Active Ingredient | alglucosidase alfa |
| Drug Class | Pompe disease |
| Manufacturer | Sanofi Genzyme |
| Dosage Forms | IV infusion, 50mg vial for reconstitution |
| Medical Code | A16AB07 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 7 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT00158600 |
| Countries | 7 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations8 Validated Nodes