OSLO, Norway, March 17, 2026 — Norway’s national “New Methods” decision forum has issued a positive recommendation for the reimbursement of HEMGENIX® (etranacogene dezaparvovec), marking a major step toward expanding access to this AAV-based gene therapy for patients with haemophilia B.
The decision paves the way for finalizing a commercial agreement with Sykehusinnkjøp, Norway’s procurement authority, and represents a key milestone in the country’s adoption of one-time gene therapies for rare diseases.
Developed by CSL Behring, HEMGENIX® is the first approved one-time gene therapy in Norway for adults with severe or moderately severe haemophilia B who do not have Factor IX inhibitors.
AAV gene therapy transforming haemophilia care
HEMGENIX® is an in vivo gene therapy built on an adeno-associated virus (AAV5) vector, delivering a functional version of the Factor IX gene directly to liver cells.
This AAV-mediated approach enables continuous production of Factor IX, significantly reducing or eliminating the need for lifelong prophylactic infusions. The therapy uses the Padua variant of Factor IX, which is 5–8 times more active than the natural protein, allowing for sustained therapeutic benefit after a single infusion.
“This recommendation reflects the transformative value that AAV gene therapy can bring to patients,” said Helena Bragd, General Manager of CSL Nordics. “HEMGENIX® represents a once-in-a-lifetime treatment opportunity.”
Strong clinical evidence from HOPE-B trial
The approval is supported by long-term data from the Phase III HOPE-B trial, which demonstrated durable efficacy and safety of this AAV5-based gene therapy over five years:
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94% of patients discontinued routine Factor IX prophylaxis
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Mean Factor IX activity levels reached ~36% and remained near-normal
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~90% reduction in annualized bleeding rate (ABR)
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Significant reductions in joint and spontaneous bleeds
Importantly, the therapy showed consistent efficacy even in patients with pre-existing AAV5 neutralizing antibodies, expanding its potential patient eligibility.
HEMGENIX® was generally well tolerated, with most adverse events occurring within the first six months and no serious treatment-related safety signals reported.
Expanding access to AAV gene therapy in Europe
Norway joins a growing list of countries—including Switzerland, Germany, the UK, and Canada—that have issued positive reimbursement decisions for HEMGENIX®, reflecting increasing global recognition of the value of AAV gene therapy in rare disease treatment.
Haemophilia B, caused by mutations in the Factor IX gene, leads to spontaneous and potentially life-threatening bleeding episodes, significantly impacting quality of life. Traditional management requires frequent intravenous infusions, placing a lifelong burden on patients.
The availability of HEMGENIX® marks a paradigm shift from chronic management to potential long-term disease control through a single administration.
