BLAINJECTIONINJECTABLEPriority Review
Approved
Aug 2022
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
7
Data completeness
3/8 — Basic
Priority Review
Drug data last refreshed 19h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
XENPOZYME (olipudase alfa-rpcp) is an intravenous enzyme replacement therapy approved for Niemann-Pick disease types A and B caused by acid sphingomyelinase deficiency. It replaces the deficient enzyme to reduce accumulation of sphingomyelin in affected tissues. This is a rare genetic lysosomal storage disorder affecting a small patient population.
$5MPart D
PeakEarly-stage commercial phase with modest Medicare spending signals a niche ultra-rare disease product requiring specialized sales and patient support infrastructure.
Mechanism of Action
Mechanism of action data is being enriched from DailyMed and FDA sources. Check back soon for updated drug intelligence.
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (7)
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
Started Apr 2024
10 enrolled
Niemann-Pick DiseasesAcid Sphingomyelinase Deficiency
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
Started Jun 2022
40 enrolled
Acid Sphingomyelinase Deficiency (ASMD)
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
Started Nov 2021
3 enrolled
Acid Sphingomyelinase Deficiency
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Started Dec 2015
36 enrolled
Sphingomyelin Lipidosis
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency
Started May 2015
20 enrolled
Sphingomyelin Lipidosis
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.