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Concizumab

Phase 3

Haemophilia A and B With and Without Inhibitors | Small molecule | Hematology |Novo Nordisk A/S|Last Updated: Dec 30, 2025

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
CONTROLLEDDMC
Total Trials1
Total Enrollment153
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05135559A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without InhibitorsPHASE3 ACTIVE NOT_RECRUITING 153 — — Mar 24, 2022Nov 2, 2029Dec 30, 202591 United States, Algeria +22
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Study Endpoints
Primary Endpoints
For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes
From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

Count of episode(s)

For non-inhibitor patients treated on demand during at least the last 52 weeks prior enrolment: Number of treated spontaneous and traumatic bleeding episodes
From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)

Count of episode(s)

Secondary Endpoints
For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of all bleeding episodes (spontaneous and traumatic)
From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated spontaneous bleeding episodes
From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
For inhibitor patients with at least 26 weeks on-demand treatment during the last 52 weeks prior enrolment: Number of treated joint bleeding episodes
From start of treatment (week 0) up until the primary analysis cut-off (at least 32 weeks)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Concizumab-naïve patientsEXPERIMENTALConcizumab-naïve participants below 12 years of age at the time of consent/assent
Patients coming from compassionate useEXPERIMENTALPatients previously treated with concizumab via compassionate use, either on an individual patient basis or through the concizumab compassionate use programme NN7415-4807
Interventions
NameTypeDescription
ConcizumabDRUGParticipants in Arm 1 will be assigned to concizumab prophylaxis starting with a loading dose on treatment day 0 followed by daily injections of an individual maintenance dose. Participants in Arm 2 will be assigned to concizumab prophylaxis with daily injections of an individual maintenance dose.
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Eligibility Criteria
SexALL
Healthy VolunteersNo
Study Sites91

Inclusion Criteria: * Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study. * Diagnosis of congenital severe haemophilia A (FVIII below...

Countries:United StatesAlgeriaBosnia and HerzegovinaBulgariaCanadaEstoniaFranceGreeceIndiaItalyJapanLebanonLithuaniaMalaysiaNorth MacedoniaNorwayPolandRomaniaSouth AfricaSpainSwedenThailandTurkey (Türkiye)United Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05135559primaryCompletionDate: changed
LOWMay 24, 2026NCT05135559studyFirstPostDate: changed