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On December 9, 2021, CSL Behring and uniQure jointly announced that a pivotal phase 3 clinical trial of etranacogene dezaparvovec has achieved positive results
In patients with hemophilia B, FIX is missing or defective due to mutations in the gene encoding FIX
▲Introduction of Etranacogene dezaparvovec (picture source: uniQure official website)
This phase 3 clinical trial enrolled 54 patients with moderate to severe adult hemophilia B whose FIX activity level was lower than or equal to 2% of the normal level and required preventive FIX replacement therapy
The key results of the test show:
At 18 months after the administration, the ABR of all hemorrhages evaluated after the stable expression of FIX was reached was 1.
After 18 months of administration, the patient's FIX activity level remained stable, and the average FIX activity level reached 36.
Etranacogene dezaparvovec is generally well tolerated, and more than 80% of adverse events are mild
Reference materials:
[1] uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B.
(The original text has been deleted)
