94% of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years post-treatment. Mean factor IX activity levels were sustained at near normal levels of 37% through four years post-treatment, reinforcing the efficacy of HEMGENIX® in the treatment of haemophilia B.
CSL Behring Denmark today announced that the first Danish patients with haemophilia B were treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec). HEMGENIX® is the first one-time gene therapy to be approved in Europe for the treatment of adults with severe and moderately severe haemophilia B.
CSL Behring today announced that an outcome-based agreement has been reached with Amgros, the Danish procurement authority, for the national reimbursement of HEMGENIX® in Denmark. Denmark is the first country in the Nordic region to reimburse HEMGENIX® under such a performance-based model, in which the regions only incur costs if the gene therapy proves effective over an agreed long-term period.
CSL Behring today announced that a positive recommendation for reimbursement of HEMGENIX has been released by the Danish Medicines Council for the treatment of patients with rare blood clotting disorder, haemophilia B opening the way to finalize a commercial contract with Amgros, the Danish procurement authority.1 Denmark is the first country in the Nordics to reimburse HEMGENIX.
Den 17 april är det Världshemofilidagen som i år har fokus på rättvis tillgång till behandling för alla med blödarsjuka. CSL Behring är ett globalt bioteknikföretag som drivs av löftet att rädda och förbättra människors liv. Forskningen inom hemofili har gjort stora framsteg under de senaste åren som har lett till nya behandlingsformer.
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.
CSL är ett globalt, specialiserat bioteknikföretag som tillverkar och tillhandahåller rekombinanta och plasmabaserade terapier. CSL Behrings innovativa produkter används för behandling av immunbrist, autoimmuna sjukdomar, hereditärt angioödem samt ärftliga och förvärvade blödningssjukdomar. Vi drivs av löftet att rädda liv och förbättra livskvaliteten för människor med sällsynta och allvarliga sjukdomar. Företaget grundades för mer än 100 år sedan av Emil von Behring – den första nobelpristagaren i medicin. Idag sysselsätter CSL över 25 000 personer i ett 30-tal länder. CSL Behrings nordiska huvudkontor ligger i Danderyd.
Besöksadress: Berga Backe 2
Box 712
182 17 Danderyd