TTR Inhibitors
2 drugsAbout TTR
Transthyretin (TTR) is a carrier protein in serum and cerebrospinal fluid, transporting thyroid hormones and retinol. Its propensity to misfold and aggregate into amyloid fibrils makes it a therapeutic target.
TTR aggregation leads to conditions like transthyretin amyloid cardiomyopathy and polyneuropathy. Currently, there is no genetic evidence data available linking TTR to specific diseases.
TTR is targeted by one FDA-approved drug, VYNDAMAX, a small molecule developed by FOLDRX PHARMS. This drug falls into the 'other' therapeutic area category.
Top TTR Drugs
FOLDRX PHARMS is the only company with an approved drug targeting TTR.
The lack of competition presents a significant market opportunity for new entrants.
TTR Drug Modality Landscape
Modalities
Routes of Administration
TTR is amenable to small molecule drugs, with oral options available for convenient dosing.
Explore alternative modalities like antibodies or gene therapies to differentiate from existing treatments.
TTR Clinical Trials 36 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on TTR. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 21 | 20 | 1 | 0 | 95% |
| Phase 2 | 1 | 0 | 1 | 0 | 0% |
| Phase 3 | 8 | 6 | 1 | 1 | 86% |
| Phase 4 | 6 | 2 | 0 | 4 | 100% |
Top Sponsors
By Modality
Top Conditions
TTR Drug Approval Timeline (2019 - 2024)
VYNDAMAX was both the first and most recently approved drug, in 2019.
The recent approval and lack of subsequent drugs suggest an emerging but unsaturated market.
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Deep insights for drug target analysis
Competitive Landscape
- • 2 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: 6 (5 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 TTR. A trial counts if an approved drug acting on TTR 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.