Senaste nyheterna

First Gene Therapy for Hemophilia B, CSL’s Hemgenix, Approved by the European Commission

First Gene Therapy for Hemophilia B, CSL’s Hemgenix, Approved by the European Commission

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.

European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B

European Medicines Agency Commences Review of Novel Gene Therapy Candidate Etranacogene Dezaparvovec for People with Hemophilia B

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.

Final Analysis of Pivotal HOPE-B Study Demonstrates Durable and Sustained Therapeutic Effect of Etranacogene Dezaparvovec Gene Therapy in Hemophilia B – Data Presented at EAHAD 2022

Final Analysis of Pivotal HOPE-B Study Demonstrates Durable and Sustained Therapeutic Effect of Etranacogene Dezaparvovec Gene Therapy in Hemophilia B – Data Presented at EAHAD 2022

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.

EMA Grants Accelerated Assessment for CSL Behring’s Haemophilia B Gene Therapy

EMA Grants Accelerated Assessment for CSL Behring’s Haemophilia B Gene Therapy

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.

Dr Vuokko Nummi at Helsinki University Hospital receives the CSL Behring Nordic von Willebrand Scholarship

Dr Vuokko Nummi at Helsinki University Hospital receives the CSL Behring Nordic von Willebrand Scholarship

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 Behring Announces Closing of Global Commercialization and License Agreement with uniQure for etranacogene dezaparvovec

CSL Behring Announces Closing of Global Commercialization and License Agreement with uniQure for etranacogene dezaparvovec

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.

Kontakter

Fredrik Sjöö, MD, PhD

Fredrik Sjöö, MD, PhD

Presskontakt Head of Medical Affairs Nordic Region Talesperson för medicinska frågor CSL Behring Norden. +46 (0)8 544 966 85
Petter Olbe

Petter Olbe

Presskontakt Head of Marketing Nordic Region +46 (0)8 544 966 77

Vårt löfte är vår drivkraft

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.

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