1st hemophilia B patient in Austria receives gene therapy Hemgenix
Gene therapy uses virus to deliver a working version of F9 gene to liver cells
The first hemophilia B patient in Austria has been treated with the gene therapy Hemgenix (etranacogene dezaparvovec), according to its developer, CSL Behring.
“We are proud and grateful to offer hemophilia B patients in Austria a treatment option that has the potential to transform their lives,” Beate Natmessnig, PhD, managing director of CSL Behring Austria, said in a company press release. “This achievement is the result of outstanding regional and national collaboration among all parties involved and is a strong testament to Austria’s innovative capabilities.”
The patient received the therapy at the Comprehensive Care Center of the University Hospital of Medicine Vienna and is now one of a group of people from other countries, including France and Denmark, who’ve been given Hemgenix outside of clinical trials. The company is working with relevant stakeholders to make sure all eligible patients have access to the therapy across Europe and elsewhere.
“For the community of affected patients and their families, this represents an enormously significant milestone: the ability to treat hemophilia with gene therapy, and the fact that this option is now available in Austria is something our community has been eagerly anticipating and hoping for over decades,” said Thomas Schindl, chairman of the Austrian Hemophilia Society.
Approving Hemgenix for hemophilia B
Right now, Hemgenix is the only gene therapy marketed for hemophilia B. It uses a harmless virus to deliver a working version of the F9 gene to cells in the liver. With these genetic instructions, cells can produce factor IX, the blood clotting protein that’s missing or defective in hemophilia B. This should restore blood clotting and help prevent and control the bleeding episodes that mark hemophilia B.
Regulatory authorities in the European Union and the U.S., among others, have approved Hemgenix for certain adults with hemophilia B. The approvals were backed by data from an ongoing Phase 3 trial called HOPE-B trial (NCT03569891) that assessed the safety and efficacy of a single dose in patients with severe or moderately severe hemophilia B.
Four-year data from HOPE-B showed bleeding rates remained low, with most patients being able to discontinue routine preventive therapies. None of the participants reported adverse events related to Hemgenix.
“It is a great opportunity for people with hemophilia B to live a symptom-free life for many years,” said Cihan Ay, MD, a hematologist at the University of Vienna.
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