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Orphan Drug Designation granted for etranacogene dezaparvovec, CSL’s gene therapy for haemophilia B, by the Therapeutics Goods Administration

TGA’s designation underscores CSL’s promise to develop and deliver a truly unique portfolio of patient-focused therapies for people with rare and serious medical conditions

08 Sep 2022

Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) announced that the Therapeutic Goods Administration (TGA) has granted Orphan Drug Designation for etranacogene dezaparvovec, a late-stage gene therapy candidate developed for haemophilia B. 

Dr Andrew Nash, Senior Vice President, Head of Research and Chief Scientific Officer at CSL said the decision represented another pivotal milestone in CSL’s promise to deliver innovative treatment options that address unmet needs for people living with rare diseases. 

“For more than 35 years, we have been at the forefront of innovation in the treatment of hereditary bleeding disorders, developing an extensive portfolio of treatment options for the community and pursuing disruptive innovations that we believe can make a difference in the lives of patient groups that we have been serving for many years,” said Dr Nash. “Orphan Drug Designation for etranacogene dezaparvovec brings our CSL Behring business and our partners at uniQure closer to our goal of delivering a life-changing treatment option for Australians living with haemophilia B.”

Orphan Drug Designation is reserved for health technologies involved in treatment, prevention or diagnosis of a life-threatening or seriously debilitating and rare disorder, and which provide a significant benefit in efficacy, safety or patient care compared to current treatments. Approximately 600 people in Australia are living with haemophilia B. Having received Orphan Drug Designation, CSL will now prepare a market authorization application for submission to the TGA, seeking review for market registration approval in due course.

Loretta Croker, Executive Director and General Manager of CSL Behring Australia and New Zealand said, “We look forward to continuing to work with the relevant Australian regulators and reimbursement agencies to make this potentially transformative medicine available to haemophilia B patients as quickly as possible.” 

About Haemophilia B and Etranacogene Dezaparvovec

Haemophilia B is a rare, life-long and potentially life-threatening disease caused by a deficiency of factor IX (FIX) in the blood, which causes patients to suffer prolonged bleeding. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatment includes life-long prophylactic infusions of FIX to temporarily replace or supplement low levels of the patient’s own clotting factor. 

Etranacogene dezaparvovec has been specifically designed to address the underlying cause of haemophilia B: a faulty gene that causes a deficiency in FIX. Treating a patient with etranacogene dezaparvovec essentially places a functional FIX gene in liver cells, addressing the disease at its source, and enables a person to continually generate their own stable levels of FIX.

Clinical trials have demonstrated that a single, one-time infusion of etranacogene dezaparvovec can enable the patient's body to increase FIX production, significantly reducing the number of bleeding episodes annually. 

The pivotal HOPE-B trial is the largest gene therapy trial in haemophilia B to date and underpins the clinical development program for etranacogene dezaparvovec. In the HOPE-B trial, haemophilia B patients classified as having a severe bleeding phenotype treated with etranacogene dezaparvovec demonstrated:

  • 64% reduction in adjusted annualised bleeding rate (ABR); and
  • superiority to prophylaxis treatment at 18 months post-treatment, compared to a 6-month run-in period (p=0.0002).

The multi-year clinical development program for etranacogene dezaparvovec was led by uniQure (Nasdaq: QURE), and sponsorship of the clinical trials has transitioned to CSL Behring after it acquired global rights to commercialise the investigational treatment.

The European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for etranacogene dezaparvovec and the Food and Drug Administration has accepted CSL’s Biologics License Application for etranacogene dezaparvovec for priority review. If approved by the TGA, it could be the first gene therapy treatment option for Australia’s haemophilia B community.

About CSL 
CSL Limited (ASX: CSL; USOTC:CSLLY) is a leading global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, as well as vaccines to prevent influenza. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses, CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 30,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit and follow us on 

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