Exjade

(deferasirox)

ApprovedSmall molecule

Deferasirox is used to remove excess iron from the body in patients who receive frequent blood transfusions or have certain blood disorders that cause iron buildup, which can damage organs if left untreated.[2] Exjade is currently approved and in use across 13 indications[4].[1]

Exjade is one of 242 small molecule programs targeting Beta-Thalassemia Major. It is currently in Approved.

Indications

Total Trials

Active Trials

241

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Beta-Thalassemia MajorApproved

HemosiderosisCompleted

Anemia (phenotype)Approved

AnemiaActive, not recruiting

Rare Hereditary HemochromatosisActive, not recruiting

HemochromatosisActive, not recruiting

Liver DiseaseApproved

Myelodysplastic SyndromeCompleted

Acute Myeloid LeukemiaApproved

Beta-ThalassemiaCompleted

Beta-Thalassemia MajorApproved

Approved

HemosiderosisApproved

Completed

Anemia (phenotype)Approved

Approved

AnemiaPreclinical

Active, not recruiting

Rare Hereditary HemochromatosisApproved

Active, not recruiting

HemochromatosisApproved

Active, not recruiting

Liver DiseaseApproved

Approved

Myelodysplastic SyndromeApproved

Completed

Acute Myeloid LeukemiaApproved

Approved

Beta-ThalassemiaApproved

Completed

Showing 10 of 13 indications

Competition

Competitive Landscape

235 competitors in Acute Myeloid Leukemia

#DrugCompanyPhaseTrials

Exjade

Novartis

Approved

74 2

Idamycin

Pfizer

Phase 3

60 3

Novantrone

Pfizer

Phase 3

34 4

Xospata

Astellas

Phase 3

24 5

Fludara

Sanofi

Phase 3

18 6

Vepesid

AAvyxa

Phase 3

52 7

Vanflyta

Daiichi Sankyo

Phase 3

16 8

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

42 9

Mylotarg

WWyeth-Lederle Vaccines

Phase 3

38 10

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2027

Phase 2Recruiting

Sickle Cell Disease

NCT07023666

Clinical

Trials by Indication[1]

Thalassemia12 trials

Renal Function Among Thalassemia Patients Treated by a Oral Chelator Deferasirox

Completed·HHaEmek Medical Center, Israel··NCT01905774·36 enrolled

Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload

Completed· Novartis logo

Novartis·Phase 1·NCT00560820·11 enrolled

An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload

Completed· Novartis logo

Novartis·Phase 3·NCT00171210·506 enrolled

Show 9 more trials

Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia

Completed· Novartis logo

Novartis·Phase 4·NCT01709838·134 enrolled

Comparison of Deferasirox and Desferoxamine in Patients of β-Thalassemia Major With Iron Overload

Completed·RRESnTEC, Institute of Research·N/A·NCT06468423·142 enrolled

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Completed· Novartis logo

Novartis·Phase 2·NCT00303329·184 enrolled

Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses

Withdrawn· Novartis logo

Novartis·Phase 2·NCT01948817

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

Completed· Novartis logo

Novartis·Phase 2·NCT00873041·166 enrolled

An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old

Withdrawn· Novartis logo

Novartis·Phase 4·NCT01724138

Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients

Unknown status·KKevin H.M. Kuo, MD, MSc, FRCPC·N/A·NCT02474420·60 enrolled

A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.

Completed· Novartis logo

Novartis·Phase 2·NCT00390858·40 enrolled

Effect of Omega 3 Fatty Acids on Hepatic and Cardiac Iron Burden and Organic Function in Children and Adolescent With Transfusion Dependent Beta Thalassemia:A Randomized Controlled Trial

Completed·AAin Shams University·Phase 2·NCT07863791·100 enrolled

Myelodysplastic Syndrome5 trials

Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.

Completed· Novartis logo

Novartis·Phase 2·NCT01868477·28 enrolled

A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy.

Terminated· Novartis logo

Novartis·Phase 2·NCT02663752·1 enrolled

Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study

Terminated· Novartis logo

Novartis·Phase 4·NCT01326845·12 enrolled

Show 2 more trials

Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload

Completed· Novartis logo

Novartis·Phase 4·NCT00564941·309 enrolled

Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion Dependent Anemia or Myelodysplastic Syndrome

Terminated·FFred Hutchinson Cancer Center·Phase 2·NCT02943668·2 enrolled

Beta-Thalassemia5 trials

Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major

Unknown status·TTanta University·N/A·NCT02761395·60 enrolled

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

Completed· Novartis logo

Novartis·Phase 2·NCT00447694·30 enrolled

A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Completed· Novartis logo

Novartis·Phase 3·NCT00171171·252 enrolled

Show 2 more trials

Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)

Completed· Novartis logo

Novartis·Phase 4·NCT00171301·233 enrolled

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

Completed· Novartis logo

Novartis·Phase 2·NCT00061763·175 enrolled

Transfusional Iron Overload4 trials

ENTRUST, a 5 Year Surveillance of Children Aged 2 to <6 Years With Transfusional Iron Overload Treated With Deferasirox

Completed· Novartis logo

Novartis··NCT00466063·108 enrolled

Extension Study of Iron Chelation Therapy With Deferasirox in Patients With Transfusional Iron Overload

Completed· Novartis logo

Novartis·Phase 2·NCT00379483·66 enrolled

Study of Deferasirox for Treatment of Transfusional Iron Overload in Myelodysplastic Patients

Completed· Novartis logo

Novartis·Phase 2·NCT00110266·176 enrolled

Show 1 more trial

Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients

Completed· Novartis logo

Novartis·Phase 4·NCT00117507·24 enrolled

Sickle Cell Disease4 trials

Clinical Importance of Treating Iron Overload in Sickle Cell Disease

Terminated·—·Phase 3·NCT00981370·1 enrolled

Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients

Completed· Novartis logo

Novartis·Phase 2·NCT00110617·212 enrolled

Low Dose Iron Chelation as TReatment of Oxidative Damage in Sickle Cell Disease

Completed·—·Phase 2·NCT05392101·12 enrolled

Show 1 more trial

Early Detection and Management of Cardiac Iron Overload in Sickle Cell Disease Using Multimodal Imaging for Improved Clinical Outcomes

Recruiting·IInova Health Care Services·Phase 2·NCT07023666·100 enrolled

32 more indications

Hereditary Hemochromatosis3 trials

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH)

Terminated· Novartis logo

Novartis·Phase 2·NCT03203850·45 enrolled

Treatment of Iron Overload With Deferasirox (Exjade) in Hereditary Hemochromatosis and Myelodysplastic Syndrome

Withdrawn·HHaukeland University Hospital·Phase 2·NCT01892644

Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis

Completed· Novartis logo

Novartis·Phase 1/2·NCT00395629·49 enrolled

Hemosiderosis3 trials

Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis

Completed· Novartis logo

Novartis·Phase 4·NCT03372083·44 enrolled

Observation of Patients With Transfusional Hemosiderosis Treatment With Deferasirox

Completed· Novartis logo

Novartis··NCT01394029·120 enrolled

Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis

Completed· Novartis logo

Novartis·Phase 2·NCT00631163·102 enrolled

Iron Overload3 trials

Treatment of Iron Overload Requiring Chelation Therapy

Withdrawn· Takeda logo

Takeda·Phase 2·NCT01927913

Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload

Completed· Novartis logo

Novartis·Phase 2·NCT00600938·197 enrolled

Early and Low Dose Deferasirox (3.5 mg/kg FCT) to Suppress NTBI and LPI as Early Intervention to Prevent Tissue Iron Overload in Lower Risk MDS

Terminated·FFondazione Italiana Sindromi Mielodisplastiche-ETS·Phase 2·NCT03920657·11 enrolled

Efficacy2 trials

Safety, Tolerability, and Efficacy of Deferasirox in MDS

Completed·GGruppo Italiano Malattie EMatologiche dell'Adulto·Phase 3·NCT00469560·158 enrolled

Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients

Completed·CConsorzio per Valutazioni Biologiche e Farmacologiche·Phase 3·NCT01825512·435 enrolled

Complication2 trials

Risk Factors and Measures to Prevent Liver and Pancreas Complications in Pediatric Patients After HSCT

Completed·UUniversity of Pisa··NCT04423237·39 enrolled

Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia

Withdrawn· Novartis logo

Novartis·Phase 4·NCT02069886

Efficacy and Safety2 trials

Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload

Completed· Novartis logo

Novartis·Phase 2·NCT01254227·60 enrolled

A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload

Completed· Novartis logo

Novartis·Phase 3·NCT00171821·1,784 enrolled

Myelodysplastic Syndromes2 trials

This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.

Completed· Novartis logo

Novartis·Phase 4·NCT01250951·111 enrolled

Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study

Completed· Novartis logo

Novartis·Phase 2·NCT00940602·225 enrolled

Stem Cell Transplant2 trials

Deferasirox in Treating Patients With Iron Overload After Undergoing a Donor Stem Cell Transplant

Terminated·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT00602446·4 enrolled

Deferasirox for Treating Patients Who Have Undergone Allogeneic Stem Cell Transplant and Have Iron Overload

Terminated·—·Phase 2·NCT01159067·1 enrolled

Safety and Efficacy2 trials

Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study

Completed· Novartis logo

Novartis·Phase 2/3·NCT01033747·70 enrolled

Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload

Terminated· Novartis logo

Novartis·Phase 2·NCT01459718·32 enrolled

Tics1 trial

Effect of a Potent Inducer Rifampicin on the Pharmacokinetics of Deferasirox in Healthy Volunteers

Completed· Novartis logo

Novartis·Phase 1·NCT00419172·22 enrolled

Treatment Compliance1 trial

Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload

Completed· Novartis logo

Novartis·Phase 2·NCT02435212·224 enrolled

Porphyria Cutanea Tarda1 trial

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Completed·UUniversity of Texas Southwestern Medical Center·Phase 3·NCT00599326·10 enrolled

Endotoxemia1 trial

Effects of Iron Loading and Iron Chelation Therapy on Innate Immunity During Human Endotoxemia

Completed·RRadboud University Medical Center·N/A·NCT01349699·30 enrolled

Magnetic Resonance Imaging (MRI)1 trial

Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload

Completed· Novartis logo

Novartis·Phase 4·NCT00673608·118 enrolled

MDS1 trial

Evaluating Low-dose Deferasirox (DFX) in Patients With Low-risk MDS Resistant or Relapsing After ESA Agents

Completed·UUniversity Hospital, Grenoble·Phase 2·NCT03387475·39 enrolled

Congenital Disorders1 trial

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Completed· Novartis logo

Novartis·Phase 3·NCT00235391·1,683 enrolled

Cardiac1 trial

Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec

Completed· Novartis logo

Novartis·Phase 2·NCT00879242·20 enrolled

Allogeneic Hematopoietic Cell Transplant1 trial

Efficacy and Safety of Oral Deferasirox (20 mg/kg/d) in Pts 3 to 6 Months After Allogeneic Hematopoietic Cell Transplantation Who Present With Iron Overload

Completed· Novartis logo

Novartis·Phase 4·NCT00654589·75 enrolled

Mucormycosis1 trial

The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study

Completed·LLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center·Phase 2·NCT00419770·20 enrolled

Blood Transfusion1 trial

Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies

Completed·WWake Forest University Health Sciences·Phase 2·NCT01273766·16 enrolled

Newly Diagnosed1 trial

Deferasirox, Cholecalciferol, and Azacitidine in the Treatment of Newly Diagnosed AML Patients Over 65

Terminated·EElizabeth Henry·Phase 2·NCT02341495·4 enrolled

Phase II1 trial

Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

Completed· Novartis logo

Novartis·Phase 2·NCT02125877·173 enrolled

ESS1 trial

Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet

Completed· Novartis logo

Novartis·Phase 2·NCT02993224·148 enrolled

Trial1 trial

Exjade-Early-Trial

Terminated·UUniversity of Erlangen-Nürnberg Medical School·Phase 2·NCT01058369·2 enrolled

Hepatic Impairment1 trial

Impact of Hepatic Impairment on the Pharmacokinetics of Deferasirox.

Completed· Novartis logo

Novartis·Phase 1·NCT00432627·24 enrolled

ALS1 trial

Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food

Completed· Novartis logo

Novartis·Phase 4·NCT00845871·65 enrolled

Pilot Study1 trial

Pilot Study for Patients With Poor Response to Deferasirox

Completed·—·Phase 4·NCT00749515·15 enrolled

TED1 trial

Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation

Completed· Novartis logo

Novartis·Phase 3·NCT02720536·53 enrolled

Acute Leukemia1 trial

The Effect of Deferasirox on Response Rate of Acute Leukemia Patients Not Treated by Standard Chemotherapy Regimens

Unknown status·IIsfahan University of Medical Sciences·Phase 1·NCT02413021·40 enrolled

Allogeneic Hematopoietic Stem Cell Transplant Recipient1 trial

Open-Label Single-Arm Pilot Study in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload

Completed· Novartis logo

Novartis·Phase 4·NCT01335035·30 enrolled

Midazolam1 trial

Study to Investigate the Effect of Deferasirox on the Pharmacokinetics of Midazolam

Completed· Novartis logo

Novartis·Phase 1·NCT00427505·22 enrolled

Unspecified1 trial

Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both

Unknown status·WWeill Medical College of Cornell University·Phase 1/2·NCT00738413·6 enrolled

Regulatory

FDA Approval Timeline[5]

Nov 2, 2005

EXJADE

NDA NDA021882

Nov 2, 2005

EXJADE

NDA NDA021882

Apr 20, 2007

EXJADE

NDA NDA021882

Dec 21, 2007

EXJADE

NDA NDA021882

Oct 2, 2008

EXJADE

NDA NDA021882

Mar 5, 2009

EXJADE

NDA NDA021882

Apr 22, 2009

EXJADE

NDA NDA021882

Jan 28, 2010

EXJADE

NDA NDA021882

Apr 11, 2011

EXJADE

NDA NDA021882

Aug 10, 2011

EXJADE

NDA NDA021882

Sep 14, 2012

EXJADE

NDA NDA021882

Jan 23, 2013

EXJADE

NDA NDA021882

Jan 23, 2013

EXJADE

NDA NDA021882

May 29, 2013

EXJADE

NDA NDA021882

Oct 21, 2013

EXJADE

NDA NDA021882

Nov 1, 2013

EXJADE

NDA NDA021882

Mar 30, 2015

JADENU

NDA NDA206910

Jul 23, 2015

EXJADE

NDA NDA021882

Aug 12, 2016

EXJADE

NDA NDA021882

May 18, 2017

JADENU SPRINKLE

NDA NDA207968

Feb 16, 2018

EXJADE

NDA NDA021882

May 11, 2018

EXJADE

NDA NDA021882

Dec 12, 2018

EXJADE

NDA NDA021882

Dec 12, 2018

EXJADE

NDA NDA021882

May 24, 2019

EXJADE

NDA NDA021882

Jul 24, 2019

EXJADE

NDA NDA021882

Jul 23, 2020

EXJADE

NDA NDA021882

Jan 17, 2023

EXJADE

NDA NDA021882

Sources

References

[1]
ClinicalTrials.gov — 74 trial records for deferasirox. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Exjade drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — deferasirox compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for deferasirox. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA021882). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Exjade. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Novartis company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: Novartis

Patent Landscape[6]

Patent Cliff

Nov 21, 2034

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9283209Nov 21, 2034

Exjade

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q4 2027

Trials by Indication[1]

Thalassemia12 trials

Myelodysplastic Syndrome5 trials

Beta-Thalassemia5 trials

Transfusional Iron Overload4 trials

Sickle Cell Disease4 trials

Hereditary Hemochromatosis3 trials

Hemosiderosis3 trials

Iron Overload3 trials

Efficacy2 trials

Complication2 trials

Efficacy and Safety2 trials

Myelodysplastic Syndromes2 trials

Stem Cell Transplant2 trials

Safety and Efficacy2 trials

Tics1 trial

Treatment Compliance1 trial

Porphyria Cutanea Tarda1 trial

Endotoxemia1 trial

Magnetic Resonance Imaging (MRI)1 trial

MDS1 trial

Congenital Disorders1 trial

Cardiac1 trial

Allogeneic Hematopoietic Cell Transplant1 trial

Mucormycosis1 trial

Blood Transfusion1 trial

Newly Diagnosed1 trial

Phase II1 trial

ESS1 trial

Trial1 trial

Hepatic Impairment1 trial

ALS1 trial

Pilot Study1 trial

TED1 trial

Acute Leukemia1 trial

Allogeneic Hematopoietic Stem Cell Transplant Recipient1 trial

Midazolam1 trial

Unspecified1 trial

FDA Approval Timeline[5]

References

Drug Info

Quick Facts

Patent Landscape[6]

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