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About GATA-1

GATA-1 is a transcription factor that binds DNA to control gene expression, crucial for red blood cell and megakaryocyte development. It regulates blood cell production, making it a key target for therapeutic intervention.

2
Approved Drugs
2
Companies
8
Indications
1
Therapeutic Areas
Broadest Approval
ANAGRELIDE HYDROCHLORIDE
Cipla
4
approved indications

Top GATA-1 Drugs

AGRYLIN
Takeda
4 indications · 1997
ANAGRELIDE HYDROCHLORIDE
Cipla
4 indications · 2005
🏢

The competitive landscape includes Cipla and Takeda, each with approved drugs targeting GATA-1.

GATA-1 Drug Modality Landscape

Modalities

Small molecule
2
100%

Routes of Administration

💊 Oral
2
100%
💡

GATA-1 is amenable to small molecule drugs, with oral options available for convenient dosing.

Focus on small molecules presents an opportunity to explore alternative modalities like antibodies or gene therapies.

Oral option available Small molecules only

GATA-1 Clinical Trials 6 trials

Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on GATA-1. Source: ClinicalTrials.gov. How we count

6
Total Trials
2
Active
2
Completed
50%
Completion Rate

Completion by Phase

Phase Total Completed Stopped Active Completion
Phase 2 2 0 2 0 0%
Phase 3 4 2 0 2 100%

Top Sponsors

Shire 2 100%
Merck Sharp & Dohme LLC 1
PharmaEssentia 1
Incyte Corporation 1 0%
French Innovative Leukemia O... 1 0%

By Modality

Small molecule
6 50%
Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)

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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: 5 (4 companies)
  • • Completion rates by condition
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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 GATA-1. A trial counts if an approved drug acting on GATA-1 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.