ACVR1 Inhibitors
2 drugsAbout ACVR1
ACVR1, or Activin A Receptor Type 1, is a protein kinase receptor involved in signaling pathways crucial for cell growth and differentiation as part of the TGF-β superfamily. It regulates cell development and its dysregulation can contribute to disease.
ACVR1 is a therapeutic target, though no genetic evidence currently links it directly to specific diseases. The existence of two FDA-approved drugs targeting ACVR1 underscores its importance in drug development.
ACVR1 is targeted by two FDA-approved small molecule drugs: VONJO (SOBI) and OJJAARA (GSK). These drugs are approved for 'other' therapeutic areas, indicating potential for expansion into new indications.
Top ACVR1 Drugs
The ACVR1 competitive landscape includes SOBI and GSK, each with an approved drug.
The limited number of companies suggests relatively low competition, potentially easing market entry for new ACVR1-targeting therapies.
ACVR1 Drug Modality Landscape
Modalities
Routes of Administration
ACVR1 is amenable to small molecule drugs, with oral options available for convenient dosing.
The exclusive use of small molecules presents an opportunity to explore alternative modalities like antibodies or biologics.
ACVR1 Clinical Trials 69 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on ACVR1. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 31 | 12 | 6 | 13 | 67% |
| Phase 2 | 28 | 3 | 9 | 16 | 25% |
| Phase 3 | 8 | 4 | 3 | 1 | 57% |
| Phase 4 | 2 | 1 | 0 | 1 | 100% |
Top Sponsors
By Modality
Top Conditions
ACVR1 Drug Approval Timeline (2022 - 2023)
The first ACVR1-targeting drug was approved in 2022, with the most recent approval in 2023.
Recent approvals indicate growing interest, but a short approval span suggests the field is still nascent with room for innovation.
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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: 34 (9 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 ACVR1. A trial counts if an approved drug acting on ACVR1 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.