The American Journal of Managed Care

AAV5 Gene Therapy for Severe Hemophilia A Successfully Enables FVIII Production

The phase 3 GENEr8-1 study demonstrated that valoctocogene roxaparvovec, an AAV5-vectored gene therapy, effectively enabled endogenous FVIII production in adults with severe hemophilia A without ...
The American Journal of Managed Care

Subcutaneous OCTA101 Trial in Severe Hemophilia A Halted Due to Safety Concerns

In a trial assessing subcutaneous OCTA101 in patients with severe hemophilia A, promising results in pharmacokinetics and efficacy were overshadowed by safety concerns. In a phase 1/2 trial assessing ...
Nasdaq

U.S. FDA Approves Pfizer’s HYMPAVZI™ (marstacimab-hncq) for the Treatment of Adults and Adolescents with Hemophilia A or B Without Inhibitors

HYMPAVZI’s approval is based on Phase 3 study results demonstrating substantial bleed reduction compared to routine prophylaxis and on-demand treatment in eligible patients with hemophilia A or B ...
Morningstar

Denecimig (Mim8) significantly reduced annualized bleeding rate in people with hemophilia A, regardless of inhibitor status, in phase 3 data published in NEJM

PLAINSBORO, N.J. and BAGSVÆRD, Denmark, April 29, 2026 /PRNewswire/ -- Today, the New England Journal of Medicine (NEJM) published 26-week results from the phase 3 FRONTIER2 trial evaluating the ...
Business Wire

Pfizer Announces Positive Topline Phase 3 Results for HYMPAVZI™ in Hemophilia A or B with Inhibitors

Study demonstrates superiority, showing both statistically significant and clinically meaningful reduction in annualized bleeding rate with a generally well-tolerated safety profile compared to ...
Monthly Prescribing Reference

Alhemo Indication Expanded to Include Hemophilia A/B Without Inhibitors

Credit: Novo Nordisk. Concizumab is a tissue factor pathway inhibitor antagonist that enhances factor Xa production during the initiation phase of coagulation. Findings showed in patients with ...
Monthly Prescribing Reference

Marstacimab Significantly Reduces Bleeding Rates in Hemophilia A, B With Inhibitors

Marstacimab significantly reduced ABR by 93% vs on-demand treatment over 12 months. Topline data were announced from a phase 3 trial evaluating marstacimab for adults and adolescents living with ...
Finanznachrichten

Novo Nordisk: Denecimig (Mim8) significantly reduced annualized bleeding rate in people with hemophilia A, regardless of inhibitor status, in phase 3 data published in NEJM

PLAINSBORO, N.J. and BAGSVÆRD, Denmark, April 29, 2026 /PRNewswire/ -- Today, the New England Journal of Medicine (NEJM) published 26-week results from the phase 3 FRONTIER2 trial evaluating the ...