Acid sphingomyelinase Inhibitors
1 drugsAbout Acid sphingomyelinase
Acid sphingomyelinase (ASM), also known as sphingomyelin phosphodiesterase 1, is an enzyme that breaks down sphingomyelin into ceramide and phosphorylcholine. It plays a crucial role in lipid metabolism within cell membranes.
ASM inhibition has shown promise in treating conditions where ceramide accumulation contributes to disease pathology. Currently, there is no genetic evidence data available linking ASM to specific diseases.
There is one FDA-approved drug, XENPOZYME (Sanofi), targeting ASM for a single indication. This drug is a biologic (other) and was first approved in 2022.
Top Acid sphingomyelinase Drugs
Sanofi is the only company with an approved drug targeting ASM.
Low competition may indicate high barriers to entry or limited market potential.
Acid sphingomyelinase Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets Acid sphingomyelinase, using enzyme modality.
The lack of modality diversity suggests an opportunity for small molecule or alternative biologic approaches.
Acid sphingomyelinase Clinical Trials 3 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Acid sphingomyelinase. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1 | 1 | 0 | 0 | 100% |
| Phase 2 | 2 | 2 | 0 | 0 | 100% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Acid sphingomyelinase Drug Approval Timeline (2022 - 2022)
The first and only drug targeting ASM was approved in 2022.
Recent approval suggests a growing interest in ASM as a therapeutic target, but market saturation is not yet a concern.
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Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 1 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 1-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 2 (2 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on Acid sphingomyelinase. A trial counts if an approved drug acting on Acid sphingomyelinase is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.