ALAS1 mRNA Inhibitors
1 drugsAbout ALAS1 mRNA
ALAS1 mRNA encodes aminolevulinate synthase 1, the first enzyme in porphyrin biosynthesis, essential for heme production. It catalyzes the condensation of glycine and succinyl CoA to form delta-aminolevulinic acid, a precursor to heme.
ALAS1 mRNA is a therapeutic target because disruptions in heme biosynthesis can lead to various disorders. Currently, there is no genetic evidence available linking ALAS1 to specific diseases, but its role in heme synthesis makes it a compelling target.
ALAS1 mRNA is targeted by one FDA-approved drug, GIVLAARI, a siRNA therapeutic developed by ALNYLAM PHARMS INC. This drug is used in a therapeutic area classified as 'other'.
Top ALAS1 mRNA Drugs
ALNYLAM PHARMS INC is the only company with an approved drug targeting ALAS1 mRNA.
The lack of competition suggests high barriers to entry or limited perceived market opportunity.
ALAS1 mRNA Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets ALAS1 mRNA, using sirna modality.
The exclusive use of siRNA presents an opportunity to explore alternative modalities like small molecules or antibodies.
ALAS1 mRNA Clinical Trials 3 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on ALAS1 mRNA. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 2 | 2 | 0 | 0 | 100% |
| Phase 3 | 1 | 1 | 0 | 0 | 100% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
ALAS1 mRNA Drug Approval Timeline (2019 - 2019)
The first and most recent drug approval for ALAS1 mRNA was GIVLAARI in 2019.
The recent approval suggests a growing interest in ALAS1 mRNA as a target, but market saturation is not yet a concern.
Pro Intelligence Preview
Deep insights for drug target analysis
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: 1 (1 company)
- • 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 ALAS1 mRNA. A trial counts if an approved drug acting on ALAS1 mRNA 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.