Fanconi Anemia

Hematology
2
Pipeline Programs
3
Companies
4
Clinical Trials
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
2
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Rocket Pharmaceuticals
1 program
1
Bone Marrow AspirationPhase 11 trial
Active Trials
NCT01331018Terminated3Est. Feb 2024
Amgen
AmgenTHOUSAND OAKS, CA
1 program
1
EtanerceptPhase 11 trial
Active Trials
NCT00965666Completed3Est. Oct 2010
Knight Therapeutics
Knight TherapeuticsQC - Montréal
2 programs
biologic sample preservation procedureN/A1 trial
microarray analysisN/A1 trial
Active Trials
NCT00899522Completed8Est. May 2013
NCT00896740Terminated90Est. Sep 2007

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Rocket PharmaceuticalsBone Marrow Aspiration
AmgenEtanercept
Knight Therapeuticsbiologic sample preservation procedure
Knight Therapeuticsmicroarray analysis

Clinical Trials (4)

Total enrollment: 104 patients across 4 trials

NCT01331018Rocket PharmaceuticalsBone Marrow Aspiration

Gene Therapy for Fanconi Anemia

Start: Feb 2012Est. completion: Feb 20243 patients
Phase 1Terminated
NCT00965666AmgenEtanercept

Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

Start: Oct 2005Est. completion: Oct 20103 patients
Phase 1Completed
NCT00899522Knight Therapeuticsbiologic sample preservation procedure

Tissue Sample Collection From Patients With Fanconi Anemia

Start: Aug 2005Est. completion: May 20138 patients
N/ACompleted
NCT00896740Knight Therapeuticsmicroarray analysis

Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy Participants

Start: Mar 2002Est. completion: Sep 200790 patients
N/ATerminated

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 companies competing in this space

Related Indications

Anemia(92)Myelofibrosis(21)Polycythemia Vera(16)Primary Immune Thrombocytopenia(15)Primary Myelofibrosis(14)Immune Thrombocytopenia(12)Thrombocytopenia(11)Beta-Thalassemia(10)Aplastic Anemia(7)Anemia of Chronic Kidney Disease(7)