The Most Expensive Gene Therapy Approved by the FDA at $3.5M for Hemophilia B

Anna Osborne
23 November 2022
Clinical Trials
Gene Therapy
Regulatory & Standards
The global biotechnology leader CSL, King of Perussia U.S., has announced that the FDA has approved its gene therapy product HEGENIX – the first, and only one time, treatment for criteria fitting adults with Hemophilia B, set at a list price of $3.5m.  

HEGENIX will be available to adults with Hemophilia B who are currently using factor IX prophylaxis therapy, or, have current or historical life-threatening haemorrhage, or, have serious and spontaneous bleeding.

Hemophilia B arises from a single gene defect and causes the insufficient production of clotting factor IX resulting in a lifelong bleeding disorder.

Current treatments for this rare genetic disorder, including factor IX replacement therapy, mean patients are faced with lifelong infusions, as well as spontaneous bleeding, limited mobility, joint damage and serve pain.

As a one-time therapy HEGENIX removes the need for continual blood infusions and reduces the risk of spontaneous bleeding. It works by allowing the patients cellular machinery to produce long-term, stable levels of factor IX. The genetic instructions remain only in the target cells and do not integrate into the patient’s DNA.

“Over the years we have seen a variety of advancements for the hemophilia community, but gene therapy is the first treatment option to offer those living with hemophilia B–and caregivers–the possibility of freedom from the need for regular, ongoing infusions,” adds Kim Phelan, COO of The Coalition for Hemophilia B.

How are you enjoying this news article? Let us know your thoughts, here >>

The FDA’s approval comes following results from HEGENIX’s multi-year Phase III trial, HOPE-B, ran by CSL’s partner uniQure. The sponsorship of this on-going clinical trial was transferred to CSL in its acquirement of the global rights to commercialize HEMGENIX.

Results from HOPE-B, the largest gene therapy trial in hemophilia B so far, demonstrated that at six months’ post infusion patients in the study produced a mean factor IX activity of 39%, decreasing to 36.7% at 24 months post infusion.

Further results showed that the mean adjusted annualized bleeding rate (ABR) for all bleeds reduced in the cohort by 54%, and that 94% (51 out of 54) of patients discontinued factor IX prophylaxis and remained prophylaxis-free.

No serious adverse reactions have been reported during this trial of HEGENIX and no inhibitors to factor IX were reported.

 “HEMGENIX is unique in its approach to increasing mean factor IX activity and hemostatic protection in those with hemophilia B, and today’s approval could fundamentally transform the treatment paradigm for this life-long condition,” said Dr. Steven Pipe, Lead Investigator in the HOPE-B study.

Source: CSL Press Release

While we have you…

We are continuously working to better optimize our content for you, our audience. Let us know how we’re doing by ticking the box below, or send us your feedback directly to