TTR mRNA Inhibitors
2 drugsAbout TTR mRNA
Transthyretin mRNA (TTR mRNA) encodes for transthyretin protein, which is primarily synthesized in the liver. The TTR protein transports thyroxine and retinol (vitamin A) in the blood.
TTR mRNA is a druggable target for modulating transthyretin protein production, offering a therapeutic avenue when aberrant protein levels or misfolded forms contribute to disease. There is currently no genetic evidence data available for this target.
Two FDA-approved drugs, ONPATTRO and AMVUTTRA, target TTR mRNA using siRNA modalities. These drugs, developed by Alnylam Pharmaceuticals, address therapeutic areas outside the common categories of diabetes, cancer, or autoimmune diseases.
Top TTR mRNA Drugs
Alnylam Pharmaceuticals is the only company with approved drugs targeting TTR mRNA.
High market concentration indicates potential entry barriers; strategic partnerships or novel approaches may be necessary to compete.
TTR mRNA Drug Modality Landscape
Modalities
Routes of Administration
TTR mRNA requires biologic approaches (sirna), likely due to its structure or location.
Consider exploring alternative modalities to differentiate from existing therapies and potentially improve delivery or efficacy.
TTR mRNA Clinical Trials 8 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on TTR mRNA. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 2 | 1 | 1 | 0 | 0 | 100% |
| Phase 3 | 7 | 4 | 0 | 3 | 100% |
Top Sponsors
By Modality
TTR mRNA Drug Approval Timeline (2018 - 2022)
The first drug targeting TTR mRNA was approved in 2018 (ONPATTRO), with the most recent approval in 2022 (AMVUTTRA).
Relatively recent entry suggests market growth potential, but also a need to establish long-term efficacy and safety.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 2 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-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 TTR mRNA. A trial counts if an approved drug acting on TTR 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.