Alglucerase

DB00088ApprovedWithdrawnProtein Based TherapiesHydrolytic Lysosomal Glucocerebroside-specific Enzyme

Alglucerase is a form of recombinant beta-glucocerebrosidase enzyme used to replace the deficient endogenous enzyme in the treatment of Gaucher disease. Human Beta-glucocerebrosidase or Beta-D-glucosyl-N-acylsphingosine glucohydrolase E.C.

Mechanism: Alglucerase catalyzes the hydrolysis of the glycolipid, glucocerebroside, to glucose and ceramide as part of the normal degradation pathway for membrane lipids.
Curator reviewed
Primary indication: Alglucerase is indicated for use as a long-term enzyme replacement therapy in patients with Type I Gaucher disease who exhibit signs and symptoms that are severe enough to result in moderate-to-severe anemia, thrombocytopenia, bone disease,...Curator reviewed
Formula / weight: C₂₅₃₂H₃₈₅₄N₆₇₂O₇₁₁S₁₆ · 55597.4 Da
First approval: United States, 1991

Resolves to

Compound

UNII27T56C7KK0CAS143003-46-7ChEMBLCHEMBL1201633

Clinical / RWD

RxNorm46049ATCA16AB01PharmGKBPA164748093

What you can answer from here — as of July 17, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action

Listed above

1Clinical trialPhase, status and sponsor resolved per trial

3Marketed productsAcross 2 countries and 2 labellers

1ATC codeIncluding every combination product, plus 9 drug categories

4+ReferencesStructured and connected to the statements they support

Which companies are running trials of Alglucerase, in which indications and phases, and which of those programs are still active?

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