Home » EU Authorizes CSL’s Gene Therapy for Hemophilia B
EU Authorizes CSL’s Gene Therapy for Hemophilia B
The European Commission has granted a conditional marketing authorization for CSL’s Hemgenix (etranacogene dezaparvovec) as the first EU-approved gene therapy for treatment of Hemophilia B.
The authorization is for adult patients with moderately severe and severe Hemophilia B, a rare genetic bleeding disorder caused by a deficiency of the Factor IX protein.
CSL’s application was supported by results from a clinical trial that demonstrated increases in Factor IX activity and a significant reduction in the annualized bleed rate of treated patients.
Hemgenix received FDA’s approval in November 2022.
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