Subcutaneous Concizumab Prophylaxis in Patients with Hemophilia A or B with Inhibitors: Efficacy and Safety Results By Hemophilia Subtype from the Phase 3 Explorer7 Trial

Frei-Jones, Melissa; Cepo, Katarina; d’Oiron, Roseline; Goh, Ai Sim; Mathias, Mary; Odgaard-Jensen, Jan

doi:10.1182/blood-2022-166522

Cited by 4 publications

(5 citation statements)

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“…53 The estimated mean (95% CI) ABR for treated spontaneous and traumatic bleeds was 1.6 (0.9-2.8) for participants with haemophilia A with inhibitors receiving concizumab in the study. 53 In addition, results from the Phase 3 explorer8 study in participants with haemophilia without inhibitors demonstrated that concizumab prophylaxis was superior to no prophylaxis, with an estimated mean (95% CI) ABR of 2.7 (1.6-4.6) for those on concizumab compared with 19.3 (11.3-33.0) for participants not receiving prophylaxis. 54 However, non-inferiority of concizumab to previous prophylaxis was not confirmed and the median (IQR) ABR for participants with haemophilia A on prior prophylaxis was 2.2 (0.8-6.2) versus 2.3 (0.0-4.7) for concizumab prophylaxis.…”

Section: Investigational Non-factor Replacement Therapiesmentioning

confidence: 89%

“…Concizumab is dosed subcutaneously daily, and marstacimab is dosed subcutaneously once weekly 50,51 . Results from the Phase 3 explorer7 study in participants with haemophilia with inhibitors reported concizumab was well‐tolerated, with no thrombotic events following restart of treatment with revised dosing 53 . The estimated mean (95% CI) ABR for treated spontaneous and traumatic bleeds was 1.6 (0.9–2.8) for participants with haemophilia A with inhibitors receiving concizumab in the study 53 .…”

Section: Non‐factor Replacement Therapies and Fviii Equivalencementioning

confidence: 99%

“…Included 63 patients with severe haemophilia A (median [range] age, 28 7–59 years) receiving tertiary prophylaxis.…”

Section: Rationale For Aiming For Higher Fviii Activity Levelsmentioning

confidence: 99%

“…Results from the Phase 3 explorer7 study in participants with haemophilia with inhibitors reported concizumab was well‐tolerated, with no thrombotic events following restart of treatment with revised dosing 53 . The estimated mean (95% CI) ABR for treated spontaneous and traumatic bleeds was 1.6 (0.9–2.8) for participants with haemophilia A with inhibitors receiving concizumab in the study 53 . In addition, results from the Phase 3 explorer8 study in participants with haemophilia without inhibitors demonstrated that concizumab prophylaxis was superior to no prophylaxis, with an estimated mean (95% CI) ABR of 2.7 (1.6–4.6) for those on concizumab compared with 19.3 (11.3–33.0) for participants not receiving prophylaxis 54 .…”

Section: Non‐factor Replacement Therapies and Fviii Equivalencementioning

confidence: 99%

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Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Malec,

Matino

2023

Haemophilia

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IntroductionThe standard of care in severe haemophilia A is prophylaxis, which has historically aimed for a factor VIII (FVIII) trough level of ≥1%. However, despite prophylactic treatment, people with haemophilia remain at risk of bleeds that have physical and quality of life implications, and that impact everyday life.AimThe aim of this review was to evaluate evidence supporting the relationship between targeting higher FVIII activity levels with prophylaxis and improved outcomes in people with haemophilia A.MethodsWe conducted a narrative review that defined the unmet needs and treatment goals in people with haemophilia A, evaluated evidence to support targeting higher FVIII activity levels, and highlighted therapies that may support higher and sustained FVIII activity levels and improved outcomes for people with haemophilia A.ResultsDespite recent advances in treatment, unmet needs remain, and people with haemophilia continue to experience joint and functional impairment, acute and chronic pain, and poor mental health. All these negatively impact their health‐related quality of life. Evidence suggests that FVIII activity levels of up to 50% may be needed to achieve a near‐zero joint bleed rate. However, achieving high FVIII activity levels with current standard and extended half‐life (EHL) FVIII replacement therapies is associated with a high treatment burden. Innovative treatment options may provide high sustained FVIII activity levels and improved patient outcomes.ConclusionEvidence suggests that FVIII activity levels in people with haemophilia A should be sustained at higher levels to improve joint and patient outcomes and enable progression towards health equity.

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Section: Investigational Non-factor Replacement Therapiesmentioning

confidence: 89%

Section: Non‐factor Replacement Therapies and Fviii Equivalencementioning

confidence: 99%

“…Included 63 patients with severe haemophilia A (median [range] age, 28 7–59 years) receiving tertiary prophylaxis.…”

Section: Rationale For Aiming For Higher Fviii Activity Levelsmentioning

confidence: 99%

Section: Non‐factor Replacement Therapies and Fviii Equivalencementioning

confidence: 99%

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Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Malec,

Matino

2023

Haemophilia

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“…6,[9][10][11][12][13][14] ABRs are approaching zero, but not zero. [9][10][11][12][13][14]16,17 The burden of treatment has been drastically reduced but persists. Treatments are not accessible to large populations of patients and where they are, patients do not always adhere to recommended treatments.…”

Section: The Hope Of Gene Therapymentioning

confidence: 99%

Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus.

Puetz¹

2023

Preprint

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After decades of investigation, gene therapy is receiving regulatory approval to treat hemophilia. However, since gene therapy investigations were initially conceived, other avenues of treatment have revolutionized the care of hemophilia. Emergent data is showing that gene therapy may not be as beneficial as hoped and more toxic than planned. At a minimum, a reassessment of risk/benefit estimate of gene therapy for hemophilia is needed.Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus

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Concizumab: First Approval

Keam

2023

Drugs

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Subcutaneous Concizumab Prophylaxis in Patients with Hemophilia A or B with Inhibitors: Efficacy and Safety Results By Hemophilia Subtype from the Phase 3 Explorer7 Trial

Cited by 4 publications

References 0 publications

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Targeting higher factor VIII levels for prophylaxis in haemophilia A: a narrative review

Emergent data influences the risk/benefit assessment of hemophilia gene therapy using recombinant adeno-associated virus.

Concizumab: First Approval

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