XENPOZYME (olipudase alfa-rpcp)
XENPOZYME is indicated for the treatment of Acid Sphingomyelinase Deficiency.
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.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2022-08-31
- Patent Cliff
- 2029
- Revenue
- $67M (Q4-2025)
- 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 )
XENPOZYME Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Clinical Trial Registry
3 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT06949358 results posted | PTA17397 2024-515304-39, 2021-004109-39 | Ph 2 | completed | A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France |
| NCT02004691 ASCEND results posted | DFI12712 U1111-1142-5963, DFI12712 | Ph 2, Ph 3 | completed | Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency |
| NCT02292654 ASCEND-Peds results posted | DFI13803 U1111-1160-6469 | Ph 1, Ph 2 | completed | Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency |
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
Indications & Usage
FDA Label (PDF)XENPOZYME is indicated for the treatment of Acid Sphingomyelinase Deficiency.
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...
Pro Intelligence Preview
Deep insights for XENPOZYME
Revenue Insights
- • Q4-2025: $67M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2029
- • Generic/biosimilar risk
Trial Analysis
- • 3 total trials
- • Stage: Declining
Competitive Landscape
- • Competitor tracking
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment