XENPOZYME
XENPOZYME (olipudase alfa-rpcp) is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for the treatment of acid sphingomyelinase deficiency (ASMD). The therapy is approved for use in both adult and pediatric patients to manage the non–central nervous system manifestations of the condition. By providing an exogenous source of the missing enzyme, it serves as a targeted treatment for this lysosomal storage disease.
How XENPOZYME Works
ASMD is caused by a deficiency in the acid sphingomyelinase (ASM) enzyme, which is responsible for degrading sphingomyelin into ceramide and phosphocholine. This deficiency leads to an intra-lysosomal accumulation of sphingomyelin and other lipids in various body tissues. XENPOZYME provides an exogenous source of ASM to replace the deficient natural enzyme and facilitate lipid degradation. The medication is not expected to cross the blood-brain barrier or impact the central nervous system manifestations of the disease.
Details
- Status
- Prescription
- First Approved
- 2022-08-31
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
XENPOZYME Approval History
What XENPOZYME Treats
1 indicationsXENPOZYME is approved for 1 conditions since its original approval in 2022. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Acid Sphingomyelinase Deficiency
XENPOZYME Boxed Warning
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with XENPOZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during XENPOZYME administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue XENPOZYME immediately and initiate appropriate medical treatment. In patients with severe h...
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with XENPOZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during XENPOZYME administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue XENPOZYME immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reactions, a desensitization procedure to XENPOZYME may be considered [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue XENPOZYME immediately and initiate appropriate medical treatment. ( 5.1 )
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
XENPOZYME FDA Label Details
ProIndications & Usage
FDA Label (PDF)XENPOZYME is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. XENPOZYME is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS Patients treated with XENPOZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily ava...
Want competitive intelligence?
See who's developing similar drugs and track their progress
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.