RepurpOS

Beta Thalassemia

Disease intelligence for Beta Thalassemia: 175 alterations and 124 ranked therapeutics from curated public databases.

Disease overview

Alterations
175
Molecular (A)
99
Clinical (B–E)
76
Direct drugs
42
Via biomarker
83
Natural agents
0
Natural products
0
Merged ranked
124
With trial/literature evidence
25

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature · Pipeline phase 6 · Updated 2026-07-18

Identifiers: MONDO: MONDO:0019402 · ORPHA: ORPHA:231249

Alterations

Showing 80 of 175 · Types A (molecular) through E (functional)

NameTypeSubtypeDirection FrequencyEvidenceSourcesLinks
Abnormal hemoglobin Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormal skeletal morphology Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormal skull morphology Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormality of iron homeostasis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormality of temperature regulation Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormality of the cardiovascular system Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormality of the dentition Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormality of the liver Pathology Pathological finding — — Moderate HPO, Monarch HPO
Abnormality of the skeletal system Pathology Pathological finding — — Moderate HPO, Monarch HPO
Adrenal insufficiency Pathology Pathological finding — — Moderate HPO, Monarch HPO
Anemia Pathology Pathological finding — — Moderate HPO, Monarch HPO
Anemia of inadequate production Pathology Pathological finding — — Moderate HPO, Monarch HPO
Anisocytosis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Arthralgia Pathology Pathological finding — — Moderate HPO, Monarch HPO
Bowing of the long bones Pathology Pathological finding — — Moderate HPO, Monarch HPO
Cholelithiasis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Cirrhosis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Decreased liver function Pathology Pathological finding — — Moderate HPO, Monarch HPO
Decreased mean corpuscular hemoglobin concentration Lab / Clinical Lab value — — Moderate HPO, Monarch HPO
Decreased mean corpuscular volume Pathology Pathological finding — — Moderate HPO, Monarch HPO
Delayed puberty Pathology Pathological finding — — Moderate HPO, Monarch HPO
Diabetes mellitus Pathology Pathological finding — — Moderate HPO, Monarch HPO
Dilated cardiomyopathy Pathology Pathological finding — — Moderate HPO, Monarch HPO
Dyspnea Pathology Pathological finding — — Moderate HPO, Monarch HPO
Elevated hepatic iron concentration Lab / Clinical Lab value — — Moderate HPO, Monarch HPO
Erythroid hyperplasia Pathology Pathological finding — — Moderate HPO, Monarch HPO
Extramedullary hematopoiesis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Failure to thrive in infancy Pathology Pathological finding — — Moderate HPO, Monarch HPO
Frontal bossing Pathology Pathological finding — — Moderate HPO, Monarch HPO
Genu valgum Pathology Pathological finding — — Moderate HPO, Monarch HPO
Growth delay Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hepatic fibrosis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hepatitis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hepatocellular carcinoma Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hepatomegaly Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hepatosplenomegaly Pathology Pathological finding — — Moderate HPO, Monarch HPO
High-output congestive heart failure Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypercoagulability Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hyperpigmentation of the skin Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hyperplasia of the maxilla Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypersplenism Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypertrophic cardiomyopathy Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypochromic microcytic anemia Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypogonadism Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypogonadotropic hypogonadism Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypoparathyroidism Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypopituitarism Pathology Pathological finding — — Moderate HPO, Monarch HPO
Hypothyroidism Pathology Pathological finding — — Moderate HPO, Monarch HPO
Increased HbA2 hemoglobin Pathology Pathological finding — — Moderate HPO, Monarch HPO
Increased total leukocyte count Pathology Pathological finding — — Moderate HPO, Monarch HPO
Irritability Pathology Pathological finding — — Moderate HPO, Monarch HPO
Jaundice Pathology Pathological finding — — Moderate HPO, Monarch HPO
Microcytic anemia Pathology Pathological finding — — Moderate HPO, Monarch HPO
Muscle weakness Functional Functional test — — Moderate HPO, Monarch HPO
Osteopenia Pathology Pathological finding — — Moderate HPO, Monarch HPO
Osteoporosis Pathology Pathological finding — — Moderate HPO, Monarch HPO
Pallor Pathology Pathological finding — — Moderate HPO, Monarch HPO
Persistence of hemoglobin F Pathology Pathological finding — — Moderate HPO, Monarch HPO
Proximal tubulopathy Pathology Pathological finding — — Moderate HPO, Monarch HPO
Pulmonary arterial hypertension Pathology Pathological finding — — Moderate HPO, Monarch HPO
Recurrent fever Pathology Pathological finding — — Moderate HPO, Monarch HPO
Reduced bone mineral density Lab / Clinical Lab value — — Moderate HPO, Monarch HPO
Reduced hemoglobin A Lab / Clinical Lab value — — Moderate HPO, Monarch HPO
Skin ulcer Pathology Pathological finding — — Moderate HPO, Monarch HPO
Splenomegaly Pathology Pathological finding — — Moderate HPO, Monarch HPO
Upslanted palpebral fissure Pathology Pathological finding — — Moderate HPO, Monarch HPO
Venous thrombosis Pathology Pathological finding — — Moderate HPO, Monarch HPO
ABCB4
ATP binding cassette subfamily B member 4
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
ADA
adenosine deaminase
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
ADD2
adducin 2
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
AHSP
alpha hemoglobin stabilizing protein
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
ANK1
ankyrin 1
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
ATP4A
ATPase H+/K+ transporting subunit alpha
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
BCL11A
BCL11 transcription factor A
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
BCL2L12
BCL2 like 12
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
BNIP3L
BCL2 interacting protein 3 like
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
C9orf72
C9orf72-SMCR8 complex subunit
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
CACNA1C
calcium voltage-gated channel subunit alpha1 C
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
CACNA1D
calcium voltage-gated channel subunit alpha1 D
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets
CACNA1F
calcium voltage-gated channel subunit alpha1 F
 Molecular Gene — — Emerging Open Targets UniProt Ensembl Open Targets

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

DrugTypePhaseEvidence ScorePrice (UK / US)Trials & literatureSourcesLinks
SIROLIMUS Small molecule Approved Strong 100 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
7 trials 2 Trial pubs
View evidence

Registry trials

  • NCT00061568 — Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias (UNKNOWN) PHASE1, PHASE2
  • NCT05357482 — Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2
  • NCT04247750 — Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) (UNKNOWN) PHASE2
  • NCT00977691 — Haploidentical PBMC Transplant for Severe Congenital Anemias (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2
  • NCT03877809 — Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (COMPLETED) PHASE2
  • NCT02105766 — Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure (ACTIVE_NOT_RECRUITING) PHASE2
  • NCT02061800 — CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2

CT.gov search · Cochrane · PubMed

 Open Targets, ChEMBL ChEMBL Open Targets
ACETAZOLAMIDE Repurposing Small molecule Approved Strong 95 UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs — ChEMBL ChEMBL Open Targets
AMITRIPTYLINE HYDROCHLORIDE Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $4–$10 / 30 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
AMPHETAMINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
BICALUTAMIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CARBACHOL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CIPROFLOXACIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CLADRIBINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb —
CLOMIPRAMINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CYCLOSPORINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
5 trials 2 Trial pubs
View evidence

Registry trials

  • NCT00029380 — Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia (COMPLETED) PHASE2
  • NCT02435901 — HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity (COMPLETED) PHASE1, PHASE2
  • NCT01917708 — Bone Marrow Transplant With Abatacept for Non-Malignant Diseases (COMPLETED) PHASE1
  • NCT04009525 — Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study (COMPLETED) PHASE4
  • NCT02342145 — Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major (COMPLETED) PHASE4

Published literature

CT.gov search · Cochrane · PubMed

 DGIdb —
DIAZEPAM Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $3–$8 / 30 tabs — ChEMBL ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
DIPYRIDAMOLE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
2 pubs
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
ELAPEGADEMASE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
EPIRUBICIN Repurposing Small molecule Approved Strong 95 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP) — ChEMBL ChEMBL Open Targets
ESOMEPRAZOLE MAGNESIUM Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
ESOMEPRAZOLE SODIUM Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
ESOMEPRAZOLE STRONTIUM Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
EXAGAMGLOGENE AUTOTEMCEL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
4 trials 3 Trial pubs
View evidence

Registry trials

  • NCT05356195 — Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) (ACTIVE_NOT_RECRUITING) PHASE3
  • NCT04208529 — A Long-term Follow-up Study in Participants Who Received CTX001 (ENROLLING_BY_INVITATION) PHASE3
  • NCT05477563 — Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease (RECRUITING) PHASE3
  • NCT03655678 — A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia (COMPLETED) PHASE2, PHASE3

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
FLUDARABINE Repurposing Small molecule Approved Strong 95 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)
8 trials 4 Trial pubs
View evidence

Registry trials

  • NCT00578292 — Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO (TERMINATED) NA
  • NCT02435901 — HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity (COMPLETED) PHASE1, PHASE2
  • NCT02179359 — Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies (TERMINATED) NA
  • NCT00408447 — Stem Cell Transplant in Sickle Cell Disease and Thalassemia (UNKNOWN) PHASE2
  • NCT01962415 — Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (RECRUITING) PHASE2
  • NCT03171831 — Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major (UNKNOWN) PHASE4
  • NCT01049854 — CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant (COMPLETED) PHASE2
  • NCT00034528 — Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia (TERMINATED) PHASE2

Published literature

CT.gov search · Cochrane · PubMed

 DGIdb —
FLUMAZENIL Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
HYDROCORTISONE BUTYRATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb —
HYDROGEN PEROXIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
1 Trial pubs
View evidence

Published literature

CT.gov search · Cochrane · PubMed

 DGIdb —
IMATINIB Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
1 pubs
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
IMIPRAMINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
INDAPAMIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
INDOMETHACIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
LANSOPRAZOLE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
LEVODOPA Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb —
MAFENIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
MARALIXIBAT Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
MECAMYLAMINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
METHAZOLAMIDE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
METHOTREXATE Repurposing Small molecule Approved Strong 95 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP) — DGIdb —
METHOXSALEN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
METOPROLOL Repurposing Small molecule Approved Strong 95 UK £2–£4 / 28 tabs · US $4–$9 / 30 tabs — ChEMBL ChEMBL Open Targets
MIANSERIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
MITOMYCIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
NALIDIXIC ACID Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
NICOTINE Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
NORFLOXACIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
ODEVIXIBAT Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
OFLOXACIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
OMEPRAZOLE Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
1 Trial pubs 1 RCT
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
OMEPRAZOLE MAGNESIUM Repurposing Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
PANTOPRAZOLE SODIUM Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
PENTOSTATIN Repurposing Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
1 trials
View evidence

Registry trials

  • NCT02105766 — Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure (ACTIVE_NOT_RECRUITING) PHASE2

CT.gov search · Cochrane · PubMed

 DGIdb —
DrugTypePhaseEvidence ScorePrice (UK / US)Trials & literatureSourcesLinks
ACETYLCYSTEINE Small molecule Phase III Moderate 80 UK £5–£12 / 30 tabs or effervescent · US $8–$15 / 30-60 tabs (NAC) — Open Targets ChEMBL Open Targets
CARNITINE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
LEVOCARNITINE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
LIPOIC ACID, ALPHA Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
LUSPATERCEPT Biologic Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
MITAPIVAT Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
SILDENAFIL Small molecule Phase III Moderate 80 UK £2–£8 / 4–8 tabs (generic) · US $5–$15 / 10-30 tabs — Open Targets ChEMBL Open Targets
THALIDOMIDE Small molecule Phase III Moderate 80 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
ALEMTUZUMAB Biologic Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
BUSULFAN Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
CYCLOPHOSPHAMIDE Small molecule Phase II Emerging 60 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP) — Open Targets ChEMBL Open Targets
DEFEROXAMINE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
DENOSUMAB Biologic Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
FILGRASTIM Biologic Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
FLUDARABINE Small molecule Phase II Emerging 60 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP) — Open Targets ChEMBL Open Targets
HONEY Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
HYDROXYUREA Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
OMEGA-3 FATTY ACIDS Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
PENTOSTATIN Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
RUSFERTIDE Biologic Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
SAPABLURSEN Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
SIROLIMUS Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
7 trials 2 Trial pubs
View evidence

Registry trials

  • NCT00061568 — Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias (UNKNOWN) PHASE1, PHASE2
  • NCT05357482 — Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2
  • NCT04247750 — Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) (UNKNOWN) PHASE2
  • NCT00977691 — Haploidentical PBMC Transplant for Severe Congenital Anemias (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2
  • NCT03877809 — Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (COMPLETED) PHASE2
  • NCT02105766 — Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure (ACTIVE_NOT_RECRUITING) PHASE2
  • NCT02061800 — CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant (ACTIVE_NOT_RECRUITING) PHASE1, PHASE2

CT.gov search · Cochrane · PubMed

 Open Targets ChEMBL Open Targets
SODIUM 2,2-DIMETHYLBUTYRATE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
SOTATERCEPT Biologic Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
SP-420 Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
TOVINONTRINE Small molecule Phase II Emerging 60 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
ABATACEPT Biologic Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
FERRIC CARBOXYMALTOSE Small molecule Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
FERRIC OXYHYDROXIDE Small molecule Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
FERROUS SULFATE Small molecule Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
IRON ISOMALTOSIDE 1000 Small molecule Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
LOBENZARIT SODIUM Small molecule Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
OMIDUBICEL Cell therapy Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
PLERIXAFOR Small molecule Phase I Emerging 35 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
ALEFACEPT Biologic Preclinical Emerging 15 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
BOTULINUM TOXIN TYPE A Biologic Preclinical Emerging 15 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
DEFERASIROX Small molecule Preclinical Emerging 15 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
DEFERIPRONE Small molecule Preclinical Emerging 15 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
EXAGAMGLOGENE AUTOTEMCEL Gene therapy Preclinical Emerging 15 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
LENOGRASTIM Biologic Preclinical Emerging 15 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — Open Targets ChEMBL Open Targets
DrugTypePhaseEvidence ScorePrice (UK / US)Trials & literatureSourcesLinks
ACETAZOLAMIDE Repurposing via ABCB4 Small molecule Approved Strong 95 UK £3–£8 / 28–56 tabs · US $8–$15 / 30 tabs — ChEMBL ChEMBL Open Targets
AMITRIPTYLINE HYDROCHLORIDE Repurposing via ADA Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $4–$10 / 30 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
AMPHETAMINE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
BICALUTAMIDE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CARBACHOL Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CIPROFLOXACIN Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CLADRIBINE Repurposing via ADA Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb —
CLOMIPRAMINE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
CYCLOSPORINE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
5 trials 2 Trial pubs
View evidence

Registry trials

  • NCT00029380 — Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia (COMPLETED) PHASE2
  • NCT02435901 — HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity (COMPLETED) PHASE1, PHASE2
  • NCT01917708 — Bone Marrow Transplant With Abatacept for Non-Malignant Diseases (COMPLETED) PHASE1
  • NCT04009525 — Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study (COMPLETED) PHASE4
  • NCT02342145 — Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major (COMPLETED) PHASE4

Published literature

CT.gov search · Cochrane · PubMed

 DGIdb —
DIAZEPAM Repurposing via ABCB4 Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $3–$8 / 30 tabs — ChEMBL ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
DIPYRIDAMOLE Repurposing via ADA Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
2 pubs
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
ELAPEGADEMASE Repurposing via ADA Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
EPIRUBICIN Repurposing via ABCB4 Small molecule Approved Strong 95 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP) — ChEMBL ChEMBL Open Targets
ESOMEPRAZOLE MAGNESIUM Repurposing via ATP4A Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
ESOMEPRAZOLE SODIUM Repurposing via ATP4A Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
ESOMEPRAZOLE STRONTIUM Repurposing via ATP4A Small molecule Approved Strong 95 UK £1–£3 / 28 tabs · US $5–$12 / 30–90 tabs
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
ETHOXZOLAMIDE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
EXAGAMGLOGENE AUTOTEMCEL Repurposing via BCL11A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
4 trials 3 Trial pubs
View evidence

Registry trials

  • NCT05356195 — Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) (ACTIVE_NOT_RECRUITING) PHASE3
  • NCT04208529 — A Long-term Follow-up Study in Participants Who Received CTX001 (ENROLLING_BY_INVITATION) PHASE3
  • NCT05477563 — Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease (RECRUITING) PHASE3
  • NCT03655678 — A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia (COMPLETED) PHASE2, PHASE3

CT.gov search · Cochrane · PubMed

 DGIdb ChEMBL Open Targets
FLUDARABINE Repurposing via ADA Small molecule Approved Strong 95 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP)
8 trials 4 Trial pubs
View evidence

Registry trials

  • NCT00578292 — Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO (TERMINATED) NA
  • NCT02435901 — HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity (COMPLETED) PHASE1, PHASE2
  • NCT02179359 — Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies (TERMINATED) NA
  • NCT00408447 — Stem Cell Transplant in Sickle Cell Disease and Thalassemia (UNKNOWN) PHASE2
  • NCT01962415 — Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (RECRUITING) PHASE2
  • NCT03171831 — Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major (UNKNOWN) PHASE4
  • NCT01049854 — CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant (COMPLETED) PHASE2
  • NCT00034528 — Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia (TERMINATED) PHASE2

Published literature

CT.gov search · Cochrane · PubMed

 DGIdb —
FLUMAZENIL Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
HYDROCORTISONE BUTYRATE Repurposing via ADA Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb —
HYDROGEN PEROXIDE Repurposing via ADA Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
1 Trial pubs
View evidence

Published literature

CT.gov search · Cochrane · PubMed

 DGIdb —
IMATINIB Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
1 pubs
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
IMIPRAMINE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
INDAPAMIDE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
INDOMETHACIN Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
ISOSORBIDE DINITRATE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
LANSOPRAZOLE Repurposing via ATP4A Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
LEVODOPA Repurposing via ADA Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — DGIdb —
MAFENIDE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
MARALIXIBAT Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —
MECAMYLAMINE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
METHAZOLAMIDE Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
METHOTREXATE Repurposing via ADA Small molecule Approved Strong 95 Hospital / IV specialty — prices per vial or dose (often hundreds–thousands USD/GBP) — DGIdb —
METHOXSALEN Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
METOPROLOL Repurposing via ABCB4 Small molecule Approved Strong 95 UK £2–£4 / 28 tabs · US $4–$9 / 30 tabs — ChEMBL ChEMBL Open Targets
MIANSERIN Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US) — ChEMBL ChEMBL Open Targets
MITOMYCIN Repurposing via ABCB4 Small molecule Approved Strong 95 Varies — check openprescribing.net (UK) or costplusdrugs.com / GoodRx (US)
No indexed hits
View evidence

CT.gov search · Cochrane · PubMed

 DGIdb —

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.