Beta Thalassemia

Disease overview

Alterations

Molecular (A)

Clinical (B–E)

Trial outcome measures (T)

Direct drugs

Via biomarker

Natural agents

Natural products

Merged ranked

124

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature · Pipeline phase 6 · Updated 2026-10-15

Identifiers: MONDO: MONDO:0019402 · ORPHA: ORPHA:848

Alterations

34 total · Types A (molecular) through E (functional). Molecular rows are Open Targets gene–disease associations (evidence column = OT association score), not measured levels; phenotypes are HPO terms annotated directly to this disease (Monarch); type T rows are outcome measures used in completed ClinicalTrials.gov studies, not alterations of the disease.

Hide alterations without a value (no direction or frequency)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
BCL11A BCL11 transcription factor A	Molecular	Gene	—	—	OT score 0.44	Open Targets	UniProt Ensembl Open Targets
CACNA1C calcium voltage-gated channel subunit alpha1 C	Molecular	Gene	—	—	OT score 0.19	Open Targets	UniProt Ensembl Open Targets
CACNA1D calcium voltage-gated channel subunit alpha1 D	Molecular	Gene	—	—	OT score 0.19	Open Targets	UniProt Ensembl Open Targets
CACNA1F calcium voltage-gated channel subunit alpha1 F	Molecular	Gene	—	—	OT score 0.19	Open Targets	UniProt Ensembl Open Targets
ANK1 ankyrin 1	Molecular	Gene	—	—	OT score 0.09	Open Targets	UniProt Ensembl Open Targets
ATP4A ATPase H+/K+ transporting subunit alpha	Molecular	Gene	—	—	OT score 0.08	Open Targets	UniProt Ensembl Open Targets
AHSP alpha hemoglobin stabilizing protein	Molecular	Gene	—	—	OT score 0.08	Open Targets	UniProt Ensembl Open Targets
ADA adenosine deaminase	Molecular	Gene	—	—	OT score 0.08	Open Targets	UniProt Ensembl Open Targets
ADD2 adducin 2	Molecular	Gene	—	—	OT score 0.07	Open Targets	UniProt Ensembl Open Targets
C9orf72 C9orf72-SMCR8 complex subunit	Molecular	Gene	—	—	OT score 0.07	Open Targets	UniProt Ensembl Open Targets
ABCB4 ATP binding cassette subfamily B member 4	Molecular	Gene	—	—	OT score 0.07	Open Targets	UniProt Ensembl Open Targets
BNIP3L BCL2 interacting protein 3 like	Molecular	Gene	—	—	OT score 0.07	Open Targets	UniProt Ensembl Open Targets
BCL2L12 BCL2 like 12	Molecular	Gene	—	—	OT score 0.07	Open Targets	UniProt Ensembl Open Targets
Abnormal hemoglobin	Pathology	Pathological finding	—	very_frequent	HPO annotation	HPO, Monarch	HPO
Abnormal skull morphology	Pathology	Pathological finding	—	frequent	HPO annotation	HPO, Monarch	HPO
Abnormality of iron homeostasis	Pathology	Pathological finding	—	frequent	HPO annotation	HPO, Monarch	HPO
Abnormality of temperature regulation	Pathology	Pathological finding	—	frequent	HPO annotation	HPO, Monarch	HPO
Abnormality of the skeletal system	Pathology	Pathological finding	—	very_frequent	HPO annotation	HPO, Monarch	HPO
Anemia	Pathology	Pathological finding	—	very_frequent	HPO annotation	HPO, Monarch	HPO
Cholelithiasis	Pathology	Pathological finding	—	occasional	HPO annotation	HPO, Monarch	HPO
Hepatitis	Pathology	Pathological finding	—	occasional	HPO annotation	HPO, Monarch	HPO
Hepatomegaly	Pathology	Pathological finding	—	frequent	HPO annotation	HPO, Monarch	HPO
Hypertrophic cardiomyopathy	Pathology	Pathological finding	—	occasional	HPO annotation	HPO, Monarch	HPO
Hypogonadotropic hypogonadism	Pathology	Pathological finding	—	frequent	HPO annotation	HPO, Monarch	HPO
Irritability	Pathology	Pathological finding	—	frequent	HPO annotation	HPO, Monarch	HPO
Microcytic anemia	Pathology	Pathological finding	—	very_frequent	HPO annotation	HPO, Monarch	HPO
Muscle weakness	Functional	Functional test	—	frequent	HPO annotation	HPO, Monarch	HPO
Pallor	Pathology	Pathological finding	—	very_frequent	HPO annotation	HPO, Monarch	HPO
Reduced bone mineral density	Lab / Clinical	Lab value	—	frequent	HPO annotation	HPO, Monarch	HPO
Respiratory insufficiency	Pathology	Pathological finding	—	frequent	HPO annotation	HPO, Monarch	HPO
Skin ulcer	Pathology	Pathological finding	—	occasional	HPO annotation	HPO, Monarch	HPO
Splenomegaly	Pathology	Pathological finding	—	very_frequent	HPO annotation	HPO, Monarch	HPO
Thrombocytopenia	Pathology	Pathological finding	—	occasional	HPO annotation	HPO, Monarch	HPO
Venous thrombosis	Pathology	Pathological finding	—	occasional	HPO annotation	HPO, Monarch	HPO

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.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Price (UK / US)	Trials & literature	Sources	Links
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 Clinical trial publication Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2003) Clinical trial publication [Umbilical cord blood transplantation (UCBT) in thalassemia children] (Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi, 2001) 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 Clinical trial publication Early Engraftment and Immune Kinetics Following Allogeneic Transplant Using a Novel Reduced-Toxicity Transplant Strategy in Children/Adolescents with High-Risk Transfusion-Dependent Thalassemia: Early Results of the ThalFAbS Trial (Transplantation and cellular therapy, 2025) Clinical trial publication Unrelated Donor Peripheral Blood Stem Cell Transplantation for Patients with β-Thalassemia Major Based on a Novel Conditioning Regimen (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2019) Clinical trial publication [Pretreatment doses of antithymocyte globubin-fresenius for allogeneic hematopoietic stem cell transplantation for beta-thalassemia major] (Nan fang yi ke da xue xue bao = Journal of Southern Medical University, 2012) Clinical trial publication Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2003) 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 Clinical trial publication Further acatalasemia mutations in human patients from Hungary with diabetes and microcytic anemia (Mutation research, 2015) 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	—

Drug	Type	Phase	Evidence	Score	Price (UK / US)	Trials & literature	Sources	Links
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

Drug	Type	Phase	Evidence	Score	Price (UK / US)	Trials & literature	Sources	Links
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 Clinical trial publication Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2003) Clinical trial publication [Umbilical cord blood transplantation (UCBT) in thalassemia children] (Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi, 2001) 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 Clinical trial publication Early Engraftment and Immune Kinetics Following Allogeneic Transplant Using a Novel Reduced-Toxicity Transplant Strategy in Children/Adolescents with High-Risk Transfusion-Dependent Thalassemia: Early Results of the ThalFAbS Trial (Transplantation and cellular therapy, 2025) Clinical trial publication Unrelated Donor Peripheral Blood Stem Cell Transplantation for Patients with β-Thalassemia Major Based on a Novel Conditioning Regimen (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2019) Clinical trial publication [Pretreatment doses of antithymocyte globubin-fresenius for allogeneic hematopoietic stem cell transplantation for beta-thalassemia major] (Nan fang yi ke da xue xue bao = Journal of Southern Medical University, 2012) Clinical trial publication Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia (Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2003) 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 Clinical trial publication Further acatalasemia mutations in human patients from Hungary with diabetes and microcytic anemia (Mutation research, 2015) 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.

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