CTX001

Phase 2/3Completed

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Interest: 38/100

Hype Score

Development Stage

✓

Pre-clinical

✓

Phase 1

Phase 2

Phase 3

Approved

Indication / Disease

Beta-Thalassemia

Conditions

Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies

Trial Timeline

Sep 14, 2018 → Nov 13, 2025

NCT ID

NCT03655678

About CTX001

CTX001 is a phase 2/3 stage product being developed by Vertex Pharmaceuticals for Beta-Thalassemia. The current trial status is completed. This product is registered under clinical trial identifier NCT03655678. Target conditions include Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn.

What happened to similar drugs?

2 of 7 similar drugs in Beta-Thalassemia were approved

Approved (2) Terminated (0) Active (5)

✅DeferasiroxNovartisApproved

🔄deferasiroxNovartisPhase 3

🔄ICL670 + deferoxamineNovartisPhase 3

✅ICL670NovartisApproved

🔄CTX001Vertex PharmaceuticalsPhase 3

Hype Score Breakdown

Clinical

Activity

Company

Novelty

Community

Clinical Trials (6)

NCT ID	Phase	Status	Start	Completion	Indication
NCT05477563	Phase 3	Recruiting	Aug 2, 2022	Jun 9, 2027	Beta-Thalassemia
NCT05356195	Phase 3	Active	May 3, 2022	May 31, 2026	Beta-Thalassemia
NCT05329649	Phase 3	Active	May 2, 2022	May 31, 2026	Sickle Cell Disease
NCT04208529	Phase 3	Recruiting	Jan 20, 2021	Sep 30, 2039	Beta-Thalassemia
NCT03745287	Phase 2/3	Completed	Nov 27, 2018	Jul 7, 2025	Sickle Cell Disease
NCT03655678	Phase 2/3	Completed	Sep 14, 2018	Nov 13, 2025	Beta-Thalassemia

Competing Products

18 competing products in Beta-Thalassemia

See all competitors

Product	Company	Stage	Hype Score
ACE-536	Merck	Phase 2	42
Deferasirox	Novartis	Approved	43
Zoledronic acid + Placebo	Novartis	Phase 2/3	34
deferasirox	Novartis	Phase 3	40
ICL670 + deferoxamine	Novartis	Phase 3	40
Deferasirox	Novartis	Phase 2	35
Deferasirox	Novartis	Phase 2	35
ICL670	Novartis	Approved	43
Bitopertin	Roche	Phase 2	35
Luspatercept + Placebo	Bristol Myers Squibb	Phase 2	39
CTX001	Vertex Pharmaceuticals	Phase 3	44
CTX001	Vertex Pharmaceuticals	Phase 3	47
CTX001	Vertex Pharmaceuticals	Phase 3	47
Mozobil + Mozobil + Mozobil	Sanofi	Pre-clinical	26
Deferitrin (GT56-252) + desferoxamine (DFO)	Sanofi	Phase 1/2	32
REGN7999 + Placebo	Regeneron Pharmaceuticals	Phase 2	42
VIT-2763 60 mg QD + VIT-2763 60 mg BID + VIT-2763 120 mg BID + Placebo	LabCorp	Phase 2	24
VIT-2763 once a day (QD) + VIT-2763 twice a day (BID) + Placebo	LabCorp	Phase 2	32