Olipudase alfa
Orphan Drug Cold Chain RequiredFDA Approved
Description
Olipudase alfa is a recombinant human acid sphingomyelinase (ASM) enzyme. It is indicated for the treatment of non-central nervous system (CNS) manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease type A/B and type B.
Indications & Therapeutic Use
Acid Sphingomyelinase Deficiency (ASMD)
Global Availability (2 countries)
| Country | Access Route | Regulatory Pathway | Lead Time | Status |
|---|---|---|---|---|
| — | — | — | — | |
| — | — | — | — |
Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
Olipudase alfa
| Generic Name | Olipudase alfa |
| Brands | 1 brand available |
| Active Ingredient | Olipudase alfa |
| Drug Class | Acid Sphingomyelinase Deficiency (ASMD) |
| Manufacturer | Sanofi Genzyme |
| Dosage Forms | Lyophilized powder for IV infusion |
| Medical Code | A16AB14 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Required |
| Lead Time | 14 days |
| Reg. Status | FDA Approved |
| Clinical Trial | NCT02004695 |
| Countries | 2 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations3 Validated Nodes