The European Commission has granted conditional marketing authorization (CMA) for HEMGENIX® (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B in adults without a history of Factor IX inhibitors. In the ongoing clinical trial, HEMGENIX® reduced the rate of annual bleeds with a single infusion.
Marketing Authorization Application (MAA) for etranacogene dezaparvovec will be reviewed under accelerated assessment and has the potential to be the first gene therapy for patients living with hemophilia B. MAA filing is supported by the phase 3 HOPE-B study which demonstrated durable and sustained therapeutic effect after a single infusion.
Data from the largest gene therapy study in Hemophilia B to date shows that etranacogene dezaparvovec is statistically superior in reducing annualized bleeding rate compared to baseline FIX prophylactic therapy. Following a single administration of etranacogene dezaparvovec, participants maintained stable Factor IX (FIX) activity through 18 months.
CSL Behring today announced that the European Medicines Agency has granted its approval for an accelerated assessment request for etranacogene dezaparvovec Marketing Authorisation Application (MAA). Etranacogene dezaparvovec is an investigational gene therapy for people with haemophilia B, a life-threatening bleeding disorder, and is currently being studied in the Phase 3 HOPE-B clinical trial.
The CSL Behring Nordic von Willebrand Scholarship is awarded every two years. Dr Vuokko Nummi receives the 2020 scholarship for research on characterization of microcirculation and associations to angiodysplasia in von Willebrand disease.
Etranacogene dezaparvovec (AMT-061) is an investigational gene therapy that may potentially provide people with hemophilia B with years of functional levels of Factor IX, a blood-clotting protein that prevents excessive bleeding. uniQure will complete the Phase 3 HOPE-B trial and scale up manufacture for initial commercial supply under an agreed plan with CSL Behring.
Vi pratar med tjugotreåriga Sara som lever med den ovanliga blödardiagnosen ITP (immunologisk trombocytopeni) som drabbar cirka 3 av 100 000 personer varje år. Sara berättar om den okunskap hon stött på i livet och hur den påverkat henne.
Under åren har det hunnit bli 20 olika avsnitt där vi lyfter frågor, sprider kunskap och sticker hål på en del fördomar om blödarsjuka. Medverkar gör unga och gamla med blödarsjuka, föräldrar, läkare och forskare för att samtala och söka svar.
Studier har visat att 60-80 % av de som lever med blödarsjuka lider av daglig smärta och att bara 40 % är nöjda med sin smärtbehandling. Vi pratar med Eva Zetterberg från Koagulationsmottagningen i Skåne om ledsmärta och olika typer av smärtlindring.
Unique gene therapy has the potential to be one of the first to market treatments to provide potentially long-term benefits with only one dose. CSL Behring builds on legacy of delivering innovative treatment options for people with Hemophilia B.
Builds on CSL Behring’s promise and commitment to the transplant community. Deal accelerates the addition of clazakizumab, an anti-IL6 MAB currently in phase III development for treatment of chronic active antibody-mediated rejection, into CSL Behring’s portfolio.
Avsnitt 19 – Von Willebrand och ny forskning
