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.