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Abstract
Etranacogene dezaparvovec (etranacogene dezaparvovec-drlb; Hemgenix®) is an adeno-associated virus vector-based gene therapy being developed by uniQure and CSL Behring for the treatment of haemophilia B. In November 2022, etranacogene dezaparvovec was approved in the USA for the treatment of haemophilia B [congenital factor IX (FIX) deficiency] in adults who are currently using FIX prophylaxis therapy, have current or historical life-threatening haemorrhage or have repeated, serious spontaneous bleeding episodes. In December 2022, etranacogene dezaparvovec also received positive opinion in the EU for the treatment of haemophilia B. This article summarizes the milestones in the development of etranacogene dezaparvovec leading to this first approval.
Etranacogene Dezaparvovec (Hemgenix·): Key Points
An adeno-associated virus vector-based gene therapy being developed by uniQure and CSL Behring for the treatment of haemophilia B
Received its first approval on 22 November 2022 in the USA
Approved for the treatment of haemophilia B (congenital FIX deficiency) in adults who are currently using FIX prophylaxis therapy, have current or historical life-threatening haemorrhage or have repeated, serious spontaneous bleeding episodes
1Introduction
Haemophilia B is an X-linked, inherited bleeding disorder caused by a deficiency of coagulation factor IX (FIX) [1-3]. The disorder is characterized by frequent and recurrent bleeding into joints or soft tissue, leading to chronic pain, disability, and impaired quality of life. Current standard-ofcare for haemophilia B includes life-long on-demand and prophylactic replacement therapy with plasma-derived or recombinant FIX concentrates, administered as intravenous infusions. FIX replacement therapy is effective at reducing bleeding episodes and is generally well tolerated; however, it is associated with substantial patient and financial burden. In addition, life-long intravenous infusions of FIX concentrates may lead to development of neutralizing antibodies (inhibitors) against FIX, which may decrease their effectiveness [1-3].
In an attempt to overcome challenges associated with FIX replacement therapy, gene therapy is being investigated as a potential new treatment paradigm for haemophilia B [1-3]. Etranacogene dezaparvovec (etranacogene dezaparvovecdrlb; Hemgenix®) is an adeno-associated virus (AAV) vector-based gene therapy being developed by uniQure and CSL Behring for the treatment of haemophilia B [4, 5]. In May 2022, the US FDA granted priority review for etranacogene dezaparvovec for the treatment of haemophilia B [6]; prior to this, it was granted orphan drug designation in the USA in April 2019. On 22 November...