Xenpozyme FDA Approval History
Last updated by Judith Stewart, BPharm on Sep 1, 2022.
FDA Approved: Yes (First approved August 31, 2022)
Brand name: Xenpozyme
Generic name: olipudase alfa
Dosage form: Lyophilized Powder for Injection
Company: Sanofi
Treatment for: Acid Sphingomyelinase Deficiency
Xenpozyme (olipudase alfa-rpcp) is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for the treatment of non-central nervous system manifestations of acid sphingomyelinase deficiency (ASMD).
- Acid sphingomyelinase deficiency is rare, genetic, lysosomal storage disease that results from reduced activity of acid sphingomyelinase (ASM), an enzyme that breaks down the lipid sphingomyelin. Symptoms may include enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding.
- Xenpozyme works by providing an exogenous source of ASM to prevent intra-lysosomal accumulation of sphingomyelin in various tissues.
- Xenpozyme is adminstered via intravenous infusion every two weeks.
- The Xenpozyme product label carries a boxed warning for severe hypersensitivity reactions including anaphylaxis. Appropriate medical support measures should be readily available. Warnings and precautions associated with Xenpozyme include infusion-associated reactions (IARs); elevated transaminases; and risk of fetal malformations during dosage initiation or escalation in pregnancy.
- Common adverse reactions in adult patients include headache, cough, diarrhea, hypotension and ocular hyperemia.
Common adverse reactions in pediatric patients include pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue and pharyngitis.
Development timeline for Xenpozyme
Further information
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