Acid Sphingomyelinase Deficiency
1
Pipeline Programs
7
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
7 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
SanofirhASM
TakedaConfirmatory Testing
Clinical Trials (2)
Total enrollment: 100,011 patients across 2 trials
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Start: Dec 2006Est. completion: Apr 200911 patients
Phase 1Terminated
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Start: May 2021Est. completion: Aug 2029100,000 patients
N/AEnrolling By Invitation
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
7 companies competing in this space