BLAINJECTIONINJECTABLEPriority Review
Approved
Aug 2022
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
5
Data completeness
3/8 — Basic
Priority Review
Drug data last refreshed 4h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
XENPOZYME (olipudase alfa-rpcp) is an enzyme replacement therapy administered by injection for the treatment of acid sphingomyelinase (ASM) deficiency, a rare lysosomal storage disorder. The drug works by replacing the deficient ASM enzyme to reduce sphingomyelin accumulation in affected tissues. It represents a precision therapeutic approach for a ultra-rare genetic disease with limited treatment options.
$0.005BPart D
PeakEarly peak-stage orphan product with modest current spend; team is likely small, specialized, and focused on rare disease infrastructure and patient identification.
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 (5)
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
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Started Dec 2013
25 enrolled
Sphingomyelin Lipidosis
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
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.