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Etavopivat Low dose

Phase 3

Sickle Cell Disease | Small molecule | Hematology |Novo Nordisk A/S|Last Updated: Apr 24, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials6
Total Enrollment1,045
FDA Designations
No designations recorded
Clinical Trials (6)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06612268A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell DiseasePHASE3 RECRUITING 408 — — Feb 17, 2025Mar 12, 2029Apr 24, 2026172 United States, Australia +19
NCT04624659A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)PHASE3 ACTIVE NOT_RECRUITING 450 — — Jan 29, 2021Mar 19, 2027Apr 6, 2026142 United States, Canada +15
NCT05725902Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell DiseasePHASE2 COMPLETED 12 — — Mar 9, 2024Mar 9, 2026Apr 8, 20261 United States
NCT05953584A 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 StrokePHASE2 RECRUITING 27 — — Jun 20, 2023Sep 20, 2027Sep 18, 20259 India, Nigeria +1
NCT06198712A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell DiseasePHASE2 RECRUITING 95 — — Jan 12, 2023Aug 8, 2029Apr 24, 202618 Canada, France +5
NCT04987489A Study of Etavopivat in Patients With Thalassemia or Sickle Cell DiseasePHASE2 COMPLETED 53 — — Mar 28, 2022Sep 24, 2025Dec 16, 202532 United States, Canada +4
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Study Endpoints
Primary Endpoints
Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact
Baseline (week 0) to week 52

Measured as Count of events.

Hemoglobin response rate
24 Weeks

Hemoglobin response rate at Week 24 (increase of \> 1 g/dL \[\> 10 g/L\] from baseline) during the blinded treatment period

Annualized vaso-occlusive crisis
52 Weeks

Annualized vaso-occlusive crisis rate during the 52-week blinded treatment period based on adjudicated vaso-occlusive crisis review

Effect of etavopivat on cerebral blood flow (CBF)
24 weeks

Change in cerebral blood flow (CBF) assessments from baseline will be summarized with descriptive statistics by nominal study visit.

Effect of etavopivat on oxygen ejection fraction (OEF)
24 weeks

Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit.

Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
24 weeks

Change in CMRO2 assessments from baseline will be summarized with descriptive statistics by nominal study visit.

Change in the highest TAMMV in any of the L/R internal carotid artery [ICA] and L/R middle cerebral artery [MCA], whichever is highest, as measured by TCD
Baseline and Week 12
Single-dose: maximum plasma concentration (Cmax)
During the 24-week primary treatment period
Single-dose: area under the plasma concentration time curve from dosing (time 0) to time t ((AUC)0-t)
During the 24-week primary treatment period
Single-dose: area under the plasma concentration time curve from zero to time infinity (AUC0-inf)
During the 24-week primary treatment period
Steady-state maximum plasma concentration (Cmax,ss)
During the 24-week primary treatment period
Steady-state area under the concentration time curve over the dosing interval (AUCtau,ss)
During the 24-week primary treatment period
Steady-state average plasma concentration (Cavg,ss)
During the 24-week primary treatment period
Steady-state minimum plasma concentration (Cmin,ss)
During the 24-week primary treatment period
Incidence of adverse events (AEs), serious adverse events (SAEs), and AEs related to etavopivat
During the 24-week primary treatment period
Number of premature discontinuations
During the 24-week primary treatment period
Number of dose interruptions
During the 24-week primary treatment period
Number of dose reductions
During the 24-week primary treatment period
Cohorts A: Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history
12 weeks

Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history

Cohorts B: Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history
12 weeks

Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history

Cohort C: Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline)
12 weeks

Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline)

Secondary Endpoints
Change in Haemoglobin (Hb) greater than 1 grams per decilitre (g/dL)
Baseline (week 0) to week 24
Time to onset of first adjudicated Vaso-occlusive crisis (VOC)
Baseline (week 0) to week 52
Change in standardised T-score on the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 7a Scale
Baseline (week 0) to week 52
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EtavopivatEXPERIMENTALParticipants will be randomised to receive oral dose of Etavopivat.
PlaceboPLACEBO_COMPARATORParticipants will be randomised to receive oral dose of placebo.
Double blind etavopivat Low DoseEXPERIMENTALDouble blind etavopivat Low Dose
Double blind etavopivat High DoseEXPERIMENTALDouble blind etavopivat High Dose
Double blind placeboEXPERIMENTALDouble blind placebo
Open label etavopivatEXPERIMENTALOpen label etavopivat
Etavopivat with HUEXPERIMENTALParticipants will receive Etavopivat 400 mg QD orally in combination with HU. The dose of HU (mg/kg) will be stable (no more than a 20% change in dosing except for weight-based changes) during the study, in the opinion of the Investigator.
Etavopivat 400 mg daily - SCD with transfusionsEXPERIMENTALPatients with sickle cell disease on chronic red blood cell transfusions
Etavopivat 400 mg daily - Thalassemia with transfusionsEXPERIMENTALPatients with thalassemia on chronic red blood cell transfusions
Etavopivat 400 mg daily - ThalassemiaEXPERIMENTALPatients with thalassemia not on chronic red blood cell transfusions
Interventions
NameTypeDescription
EtavopivatDRUGEtavopivat will be administered orally.
PlaceboDRUGPlacebo matching Etavopivat will be administered orally.
Etavopivat Tablets Low doseDRUG200 mg once daily
Etavopivat Tablets High doseDRUG400 mg once daily
Placebo TabletsDRUGPlacebo once daily
Etavopivat TabletsDRUGSelected dose once daily
HydroxyureaDRUGParticipants will receive a stable dose of HU.
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites172

Inclusion Criteria: * Male or female. * Age 12 years or above at the time of signing the informed consent. * Confirmed diagnosis of sickle cell disease: Documentation of sickle cell disease (SCD) genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of lab...

Countries:United StatesAustraliaBelgiumBrazilCanadaColombiaFranceGhanaGreeceIndiaItalyKenyaLebanonNetherlandsNigeriaOmanSaudi ArabiaSpainTurkey (Türkiye)UgandaUnited KingdomEgyptGermany
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Competitive Landscape -Sickle Cell Disease 28 trials
CompanyTickerTrialsLead PhaseDrugs
Novo Nordisk A/S Sponsored ADR Class BNVO5PHASE3Etavopivat Low dose, Etavopivat, Etavopivat A, Etavopivat B, Etavopivat C
Novartis AG Sponsored ADRNVS4PHASE3Crizanlizumab, OTQ923, ITU512
Sanofi SA Sponsored ADRSNY2PHASE3PCV21, 20vPCV, Rilzabrutinib
Vertex Pharmaceuticals IncorporatedVRTX3PHASE3CTX001
Agios Pharmaceuticals, Inc.AGIO2PHASE2Mitapivat, Tebapivat
Pfizer Inc.PFE1PHASE2Osivelotor
Fulcrum Therapeutics, Inc.FULC2PHASE2Pociredir
Bristol-Myers Squibb CompanyBMY1PHASE1BMS-986470, Famotidine
Beam Therapeutics, Inc.BEAM2PHASE1BEAM-101, Long-Term Follow-up Study of patients who received BEAM-101
Editas Medicine, Inc.EDIT2PHASE1EDIT-301
Disc Medicine, Inc.IRON1PHASE1DISC-3405
Illumina, Inc.ILMN1 — Undisclosed
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT05725902TRIAL_REMOVED: changed
LOWMay 26, 2026NCT06198712Enrollment: 50 → 95
LOWMay 26, 2026NCT06612268primaryCompletionDate: changed
LOWMay 26, 2026NCT04624659primaryCompletionDate: changed
LOWMay 24, 2026NCT06198712studyFirstPostDate: changed
LOWMay 24, 2026NCT06612268studyFirstPostDate: changed
LOWMay 24, 2026NCT04624659studyFirstPostDate: changed
LOWMay 24, 2026NCT05725902studyFirstPostDate: changed