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Etavopivat A

Phase 3

Sickle Cell Disease | Small molecule | Hematology |Novo Nordisk A/S|Last Updated: Mar 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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment480
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06609226A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or ThalassaemiaPHASE3 RECRUITING 480Jan 10, 2025Dec 30, 2030Mar 24, 2026103 United States, Canada +15
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Study Endpoints
Primary Endpoints
Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately
Baseline (week 0 of FLORAL) up to end of study (up to week 316)

Measured as number of events.

Number of adverse reactions, reported for each indication and age group separately
Baseline (week 0 of FLORAL) up to end of study (up to week 316)

Measured as number of adverse reactions.

Secondary Endpoints
Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately
Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)
Change in VOCs, reported for each age group separately
Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
Change in hemoglobin (Hb) concentration, reported for each age group separately
Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Participants greater than or equal to (≥) 12 years old with sickle cell diseaseEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 12 years old with sickle cell disease transfusion dependentEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 12 years old with transfusion-dependent thalassaemiaEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 12 years old with non-transfusion dependent thalassaemiaEXPERIMENTALParticipants will receive an oral dose of Etavopivat A or C.
Participants ≥ 2 years to less than (<) 12 years old with sickle cell diseaseEXPERIMENTALParticipants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants \< 12 years of age will receive an oral dose of Etavopivat B.
Interventions
NameTypeDescription
Etavopivat ADRUGParticipants will receive an oral dose of Etavopivat A.
Etavopivat BDRUGParticipants will receive an oral dose of Etavopivat B.
Etavopivat CDRUGParticipants will receive an oral dose of Etavopivat C.
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Eligibility Criteria
Age Range2 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites103

Inclusion Criteria: * Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study. * Participant must have derived clinical benefit from treatment with etavopi...

Countries:United StatesCanadaEgyptFranceGermanyGhanaGreeceIndiaItalyKenyaLebanonNigeriaOmanSaudi ArabiaSpainTurkey (Türkiye)United Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06609226primaryCompletionDate: changed
LOWMay 24, 2026NCT06609226studyFirstPostDate: changed