Sickle Cell Disease

Rare Diseases
54
Pipeline Programs
28
Companies
50
Clinical Trials
10 recruiting
3
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
15
2
17
3
13
4
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody
450%
Peptide
225%
Small Molecule
113%
Cell Therapy
113%
+ 54 programs with unclassified modality

On Market (3)

Approved therapies currently available

Novartis
ADAKVEOApproved
crizanlizumab
Novartis
Selectin Blocker [EPC]injection2019
Pfizer
OXBRYTAApproved
voxelotor
Pfizer
Hemoglobin S Polymerization Inhibitor [EPC]oral2021
U
PRASUGREL HYDROCHLORIDEApproved
prasugrel
Unknown Company
oral2023

Competitive Landscape

27 companies ranked by most advanced pipeline stage

Pfizer
PfizerNEW YORK, NY
16 programs
2
2
1
2
1
Voxelotor Oral TabletPhase 41 trial
InclacumabPhase 3Monoclonal Antibody1 trial
VoxelotorPhase 31 trial
OsivelotorPhase 2/31 trial
VoxelotorPhase 21 trial
+11 more programs
Active Trials
NCT06503458Terminated52Est. Apr 2024
NCT03105271Completed60Est. Jan 2022
NCT00386048Completed50Est. Aug 2012
+13 more trials
Novartis
NovartisBASEL, Switzerland
3 programs
2
1
1
CrizanlizumabPhase 3Monoclonal Antibody1 trial
OTQ923Phase 11 trial
OTQ923Phase 11 trial
Active Trials
NCT06155500Active Not Recruiting4Est. Jan 2039
NCT04443907Terminated4Est. Jan 2025
NCT06439082Recruiting315Est. Apr 2030
Novo Nordisk
Novo NordiskBAGSVAERD DENMARK, Denmark
8 programs
5
3
EtavopivatPhase 31 trial
Etavopivat APhase 31 trial
Etavopivat Tablets Low dosePhase 31 trial
EtavopivatPhase 21 trial
EtavopivatPhase 21 trial
+3 more programs
Active Trials
NCT05725902Active Not Recruiting12Est. Feb 2026
NCT06198712Recruiting50Est. Aug 2029
NCT05953584Recruiting27Est. Sep 2027
+5 more trials
VP
3 programs
1
2
CTX001Phase 31 trial
Exa-celPhase 3Cell Therapy1 trial
CTX001Phase 2/31 trial
Active Trials
NCT03745287Completed63Est. Jul 2025
NCT05329649Active Not Recruiting13Est. May 2026
NCT05951205Not Yet Recruiting12Est. Dec 2033
DS
Daiichi SankyoChina - Shanghai
2 programs
1
1
PrasugrelPhase 31 trial
PrasugrelPhase 21 trial
Active Trials
NCT01476696Completed33Est. Nov 2012
NCT01794000Terminated341Est. Dec 2015
Eli Lilly and Company
Eli Lilly and CompanyINDIANAPOLIS, IN
2 programs
1
1
PrasugrelPhase 3
PrasugrelPhase 24 trials
Active Trials
NCT01430091Completed18Est. Oct 2011
NCT01115738Completed282Est. Nov 2011
NCT01591317Completed30Est. May 2009
+1 more trials
CRISPR Therapeutics
2 programs
1
1
CTX001Phase 3
CTX001Phase 2/3
J&
Johnson & JohnsonNEW BRUNSWICK, NJ
1 program
1
ICA-17043Phase 31 trial
Active Trials
NCT00294541Terminated51Est. Jun 2007
Sanofi
SanofiPARIS, France
1 program
1
PCV21Phase 32 trials
Active Trials
NCT07247188Recruiting100Est. Jan 2027
NCT06838000Active Not Recruiting1,268Est. Dec 2026
Cardurion Pharmaceuticals
3 programs
1
2
IMR-687Phase 21 trial
IMR-687Phase 21 trial
IMR-687Phase 11 trial
Active Trials
NCT02998450CompletedEst. Jul 2017
NCT03401112CompletedEst. Aug 2020
NCT04474314TerminatedEst. May 2022
Fulcrum Therapeutics
3 programs
2
1
PociredirPhase 21 trial
PociredirPhase 11 trial
Pociredir oral capsulePhase 11 trial
Active Trials
NCT07431398RecruitingEst. Apr 2026
NCT05169580CompletedEst. Jan 2026
NCT07401823Not Yet RecruitingEst. Jul 2030
City Therapeutics
City TherapeuticsMA - Cambridge
2 programs
1
1
HydroxyureaPhase 21 trial
AlemtuzumabPhase 1Monoclonal Antibody1 trial
Active Trials
NCT05384756Active Not RecruitingEst. Dec 2026
NCT01389024CompletedEst. May 2022
CSL Behring
CSL BehringIL - Bradley
2 programs
1
1
AnumigilimabPhase 2Monoclonal Antibody1 trial
CSL889Phase 11 trial
Active Trials
NCT04285827Completed28Est. Jul 2023
NCT07224360Recruiting63Est. Jun 2028
Design Therapeutics
1 program
1
GabapentinPhase 21 trial
Active Trials
NCT01954927CompletedEst. Jan 2018
Bausch Health
Bausch HealthQUEBEC, Quebec, Canada
1 program
1
Low Dose Rifaximin ERPhase 21 trial
Active Trials
NCT05098028CompletedEst. Sep 2023
BioMarin Pharmaceutical
1 program
1
Sapropterin DihydrochloridePhase 21 trial
Active Trials
NCT00445978Completed32Est. Jun 2009
Grifols
GrifolsNEW YORK, NY
1 program
1
Immune Globulin IntravenousPhase 1/21 trial
Active Trials
NCT01757418CompletedEst. Dec 2024
GC Biopharma
GC BiopharmaKorea - Yongin
1 program
1
SCD-Omegatex™Phase 1/21 trial
Active Trials
NCT02947100TerminatedEst. Oct 2018
ExCellThera
ExCellTheraQC - Montréal
1 program
1
ECT-001-CBPhase 11 trial
Active Trials
NCT04594031WithdrawnEst. Feb 2024
Astellas
AstellasChina - Shenyang
1 program
1
LexiscanPhase 14 trials
Active Trials
NCT03103061Completed24Est. Jun 2018
NCT01697995Unknown650Est. Dec 2016
NCT01085201Completed39Est. Mar 2013
+1 more trials
Biogen
BiogenCAMBRIDGE, MA
1 program
1
MotixafortidePhase 1Peptide1 trial
Active Trials
NCT05618301Completed10Est. Jun 2025
BioLineRx
BioLineRxIsrael - Modi’in
1 program
1
MotixafortidePhase 1Peptide
Bayer
BayerLEVERKUSEN, Germany
1 program
1
aspirinPhase 11 trial
Active Trials
NCT00178464Completed11Est. Nov 2009
Secura Bio
Secura BioNV - Las Vegas
1 program
1
panobinostatPhase 1Small Molecule1 trial
Active Trials
NCT01245179RecruitingEst. Jan 2027
Allergy Therapeutics
Allergy TherapeuticsUK - West Sussex
1 program
Near infrared spectroscopyN/A1 trial
Active Trials
NCT04031521WithdrawnEst. Sep 2023
Providence Therapeutics
1 program
Non-InterventionalN/A1 trial
Active Trials
NCT06529042Enrolling By InvitationEst. Dec 2026
Heidelberg Pharma
Heidelberg PharmaGermany - Ladenburg
1 program
Sickle-cell Disease Registry of the GPOHN/A1 trial
Active Trials
NCT03327428Recruiting1,000Est. Dec 2040

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
PfizerVoxelotor Oral Tablet
AstellasLexiscan
Vertex PharmaceuticalsExa-cel
SanofiPCV21
SanofiPCV21
Novo NordiskEtavopivat
Novo NordiskEtavopivat A
NovartisCrizanlizumab
Vertex PharmaceuticalsCTX001
PfizerInclacumab
Novo NordiskEtavopivat Tablets Low dose
PfizerVoxelotor
Daiichi SankyoPrasugrel
Johnson & JohnsonICA-17043
PfizerOsivelotor

Showing 15 of 50 trials with date data

Clinical Trials (50)

Total enrollment: 6,275 patients across 50 trials

NCT05228821PfizerVoxelotor Oral Tablet

Voxelotor Brain Oxygenation and Neurocognitive Study

Start: Aug 2023Est. completion: Aug 20230
Phase 4Withdrawn

Lexiscan(TM) Rb-82 Myocardial Perfusion PET: A Comparison With Dipyridamole Stress

Start: Dec 2008Est. completion: Sep 201232 patients
Phase 4Unknown

Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/βC Genotype

Start: Jul 2027Est. completion: Dec 203312 patients
Phase 3Not Yet Recruiting

Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease

Start: Jan 2026Est. completion: Jan 2027100 patients
Phase 3Recruiting

Study of the Safety and Immunogenicity of Catch-up Vaccination With a 21-valent Pneumococcal Conjugate Vaccine (PCV21) in Healthy Infants, Toddlers, Children, and Adolescents

Start: Feb 2025Est. completion: Dec 20261,268 patients
Phase 3Active Not Recruiting

A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

Start: Feb 2025Est. completion: Mar 2029408 patients
Phase 3Recruiting

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Start: Jan 2025Est. completion: Dec 2030480 patients
Phase 3Recruiting
NCT06439082NovartisCrizanlizumab

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)

Start: Oct 2024Est. completion: Apr 2030315 patients
Phase 3Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

Start: May 2022Est. completion: May 202613 patients
Phase 3Active Not Recruiting

A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

Start: Mar 2022Est. completion: Nov 2025242 patients
Phase 3Terminated
NCT04624659Novo NordiskEtavopivat Tablets Low dose

A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

Start: Jan 2021Est. completion: Mar 2027450 patients
Phase 3Active Not Recruiting

Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031

Start: Jun 2018Est. completion: Nov 2024179 patients
Phase 3Terminated

A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)

Start: Apr 2013Est. completion: Dec 2015341 patients
Phase 3Terminated

A Study Evaluating the Long-Term Safety of ICA-17043 in Sickle Cell Disease Patients With or Without Hydroxyurea Therapy

Start: Feb 2006Est. completion: Jun 200751 patients
Phase 3Terminated

A Phase 2/3 Study in Adult and Adolescent Participants With SCD

Start: Sep 2022Est. completion: Dec 2032389 patients
Phase 2/3Recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Start: Nov 2018Est. completion: Jul 202563 patients
Phase 2/3Completed

Open-Label Extension Study to Pioneer Study 6058-SCD-101

Start: Mar 2026Est. completion: Jul 2030
Phase 2Not Yet Recruiting

Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)

Start: Feb 2026Est. completion: Jun 202863 patients
Phase 2Recruiting

Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

Start: Mar 2024Est. completion: Feb 202612 patients
Phase 2Active Not Recruiting

Assessing Physical Function in Sickle Cell Patients Taking Voxelotor

Start: Oct 2023Est. completion: Jun 20266 patients
Phase 2Active Not Recruiting

A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke

Start: Jun 2023Est. completion: Sep 202727 patients
Phase 2Recruiting

A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

Start: Jan 2023Est. completion: Aug 202950 patients
Phase 2Recruiting
NCT05405114Novo NordiskNDec - oral decitabine-tetrahydrouridine

Research Study Investigating How Well NDec Works in People With Sickle Cell Disease

Start: Jul 2022Est. completion: Jul 202596 patients
Phase 2Completed
NCT04987489Novo NordiskEtavopivat tablets

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

Start: Mar 2022Est. completion: Sep 202553 patients
Phase 2Completed
NCT05098028Bausch HealthLow Dose Rifaximin ER

Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease

Start: Mar 2022Est. completion: Sep 2023
Phase 2Completed

A Study of IMR-687 in Subjects With Sickle Cell Disease

Start: Aug 2020Est. completion: May 2022
Phase 2Terminated

A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

Start: Jan 2018Est. completion: Aug 2020
Phase 2Completed

Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

Start: Jul 2016Est. completion: Oct 2023147 patients
Phase 2Terminated

Use of Lexiscan for Myocardial Stress Perfusion Computed Tomography With a 3rd Generation Dual Source CT System

Start: Feb 2016Est. completion: Jun 201824 patients
Phase 2Completed

Pain Management in Children and Young Adults With Sickle Cell Disease

Start: Oct 2013Est. completion: Jan 2018
Phase 2Completed

Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease

Start: Aug 2012Est. completion: May 2022
Phase 2Completed

A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease

Start: Nov 2011Est. completion: Nov 201233 patients
Phase 2Completed

Clopidogrel to Prasugrel in Acute Coronary Syndrome (ACS) Patients

Start: May 2010Est. completion: Nov 2011282 patients
Phase 2Completed
NCT00445978BioMarin PharmaceuticalSapropterin Dihydrochloride

A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease

Start: May 2007Est. completion: Jun 200932 patients
Phase 2Completed

A Trial of CS-747 (Prasugrel) Compared With Clopidogrel in Patients Undergoing Percutaneous Coronary Intervention (PCI)

Start: Apr 2003Est. completion: Jan 2004905 patients
Phase 2Completed

Omega-3 Fatty Acids in Sickle Cell Disease

Start: Jan 2018Est. completion: Oct 2018
Phase 1/2Terminated
NCT01757418GrifolsImmune Globulin Intravenous

Intravenous Gammaglobulin for Sickle Cell Pain Crises

Start: Nov 2008Est. completion: Dec 2024
Phase 1/2Completed

Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

Start: Dec 2025Est. completion: Apr 2026
Phase 1Recruiting

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Start: Apr 2024Est. completion: Jan 20394 patients
Phase 1Active Not Recruiting
NCT05618301BiogenMotixafortide

Motixafortide and Natalizumab to Mobilize CD34+ Hematopoietic Stem Cells for Gene Therapies in Sickle Cell Disease (SCD)

Start: Jul 2023Est. completion: Jun 202510 patients
Phase 1Completed

A GBT021601 ADME Microtracer Study in Healthy Volunteers

Start: Dec 2022Est. completion: Aug 20239 patients
Phase 1Completed

TMLI and Alemtuzumab for Treatment of Sickle Cell Disease

Start: Jul 2022Est. completion: Dec 2026
Phase 1Active Not Recruiting
NCT05169580Fulcrum TherapeuticsPociredir oral capsule

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir

Start: Dec 2021Est. completion: Jan 2026
Phase 1Completed

US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease

Start: Jul 2021Est. completion: Feb 2024
Phase 1Withdrawn

Safety of Single Doses of CSL889 in Adult Patients With Sickle Cell Disease

Start: May 2021Est. completion: Jul 202328 patients
Phase 1Completed

A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants

Start: Dec 2020Est. completion: Feb 2023129 patients
Phase 1Completed

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

Start: Aug 2020Est. completion: Jan 20254 patients
Phase 1Terminated

A Study of IMR-687 in Healthy Adult Volunteers

Start: Oct 2016Est. completion: Jul 2017
Phase 1Completed

A Relative Bioavailability Study of a Prasugrel Orally Disintegrating Tablet

Start: Sep 2011Est. completion: Oct 201118 patients
Phase 1Completed

Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

Start: Nov 2010Est. completion: Jan 2027
Phase 1Recruiting

Related Jobs in Rare Diseases

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

13 late-stage (Phase 3) programs — potential near-term approvals
10 actively recruiting trials targeting 6,275 patients
28 companies competing in this space