The National “New Methods” Decision Forum in Norway recommends CSL’s gene therapy HEMGENIX® for adults with severe or moderately severe haemophilia B without a history of Factor IX inhibitors

Oslo, Norway – March 17, 2026 – The National “New Methods” decision forum in Norway yesterday announced a positive recommendation for the reimbursement of HEMGENIX® (etranacogene dezaparvovec) for the treatment of patients with the rare blood clotting disorder haemophilia B, paving the way for the finalization of a commercial contract with Sykehusinnkjøp, the Norwegian procurement authority.

HEMGENIX^® is the first one-time gene therapy approved in Norway for the treatment of severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.¹

This is a key milestone in the broader paradigm shift being seen in the treatment of haemophilia B. Expanded access to HEMGENIX® can help reduce the burdens of lifetime prophylaxis and significantly improve patients quality of life and mental well-being.²

“We have been working diligently with all relevant stakeholders to enable access to HEMGENIX^®, since receiving approval from the European Commission 2023”, said Helena Bragd, General Manager of CSL Nordics, “The positive recommendation for HEMGENIX^® demonstrates the Norwegian healthcare system's recognition of the transformative value HEMGENIX^® brings to patients, their careers, and healthcare professionals as a once-in-a-lifetime therapy.

“Patients with haemophilia B can face bleeds in joints, muscles and soft tissues, leading to pain, swelling and potential long-term joint damage,” said Fredrik Sjöö, MD, PhD, Head of Medical Affairs, Nordic Region “Until now, people living with severe and moderately severe haemophilia B have required regular, ongoing treatment with intravenous Factor IX infusions, which can have a significant impact on quality of life. Availability of gene therapy represents a major step forward in providing greater freedom to patients, so they can lead a full life with fewer limitations.”

CSL is working with relevant stakeholders to enable the expansion of access to HEMGENIX^® across Europe, building on increasing positive reimbursement decisions in Switzerland, Germany, Italy Spain, Denmark, Scotland, the UK , Austria US, Canada and Saudi Arabia .^3-7

About Haemophilia B

Haemophilia B is a rare disease, and some bleeds may be life-threatening. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage.¹² The constant worry of a bleed means that their daily activities can be restricted, even for things as simple as going up and down stairs.^12-14 Current treatments for moderate to severe haemophilia B include life-long prophylactic infusions of Factor IX to temporarily replace or supplement low levels of the blood-clotting factor.¹² Many people with haemophilia find themselves continually confronted with the mental and emotional impact of managing their condition, and rarely have their minds free of haemophilia.¹³

About HEMGENIX^® (etranacogene dezaparvovec)

HEMGENIX^® is an in vivo gene therapy that reduces the rate of abnormal bleeding in eligible people with haemophilia B by enabling the body to continuously produce Factor IX, the protein that is deficient in people with the disease.¹⁵ It uses a non-infectious viral vector derived from an adeno-associated virus (AAV5).¹⁵ The AAV5 vector carries the Padua gene variant of Factor IX to the target cells in the liver, generating Factor IX proteins that are 5–8x more active than normal.¹⁶ These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA.¹⁵ Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of Factor IX.¹⁷

About the Pivotal HOPE-B Trial

The pivotal Phase III HOPE-B trial was a multinational, open-label, single-arm study to evaluate the safety and efficacy of HEMGENIX^®.¹⁸ A total of 54 adult patients with haemophilia B, classified as having moderately severe to severe haemophilia B and requiring prophylactic Factor IX replacement therapy, were enrolled in a prospective, 6-month or longer observational period. During this period, patients continued to use their current standard of care therapy to establish a baseline annual bleeding rate (ABR).¹⁸ After the 6-month lead-in period, patients received a single intravenous administration of HEMGENIX^® at the 2x10¹³gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralising antibodies (NAbs) to AAV5.¹⁸

The five-year data mark the final analysis for the HOPE-B study, but participants who consent will continue to be monitored in the IX-TEND 222-3003 extended follow-up study (NCT05962398), which will track patients for up to 15 years post-treatment.^10,11

The Phase III HOPE-B trial demonstrated the 5-year efficacy and safety of HEMGENIX^® as well as the ongoing benefit of this treatment for people living with haemophilia B, with 5-year bleed protection provided by a one-time infusion.^10,11 A total of 94% of patients (51/54) discontinued routine Factor IX prophylaxis and remained prophylaxis-free at 5 years post-treatment. HEMGENIX^® demonstrated mean Factor IX activity levels of 36.1% (n=48), which were sustained at near normal levels at 5 years post-treatment. Mean adjusted annualised bleeding rate (ABR) for all bleeds was reduced by approximately 90% vs lead-in of Factor IX prophylaxis (4.16, n=54) as compared to year five (0.40, n=51).^10,11 Additionally, joint bleeds were reduced by 93% from lead-in (mean ABR of 2.34 at lead-in to 0.16 at year five) and spontaneous bleeds were reduced by 94% (mean ABR of 1.52 during lead-in versus 0.09 during year five).^10,11 The results also showed that HEMGENIX^® is clinically effective in eligible patients with pre-existing AAV5 NAbs (up to a NAb titre of 1:898 or equivalent).^15,19

HEMGENIX^® was generally well-tolerated, with a total of 100 treatment-related adverse events (TRAEs) 5 years post-infusion, most of which occurred in the first six months post-treatment.^10,11 No serious treatment-related adverse reactions were reported.^15,19 Two deaths occurred during the trial due to non-treatment-related TRAEs: one at approximately 15 months post-dose due to cardiogenic shock and urosepsis, and another at approximately 54 months post-dose due to cardiac amyloidosis. Three previously reported serious adverse events (hepatocellular carcinoma, schwannoma and myelodysplastic syndrome) were determined to be unrelated to treatment with HEMGENIX^® by independent molecular tumour characterisation and vector integration analysis.^11,20 No inhibitors to Factor IX were reported.^11,15

Additional trial data

CSL220 (formerly AMT-060) has a similar structure to HEMGENIX^®, differing only in a single amino acid substitution in the F9 gene.²¹ In a Phase I/II study, the durability of CSL220 was assessed in two cohorts of adult patients with severe/moderately severe haemophilia B receiving different single-infusion doses.²¹ Mean Factor IX activity remained stable in both cohorts at 8 years (Cohort 1: n=3, Year 8: 4.9 IU/dL; Cohort 2: n=5, Year 8: 5.6 IU/dL), mean ABR was maintained at Year 8 (Cohort 1: 2.2; Cohort 2: 1.0) with no new safety events identified were identified during Year 8 and no patients returning to continuous Factor IX prophylaxis.²¹

In a Phase IIb study of adults (n=3) with haemophilia B receiving a single dose of HEMGENIX^®, mean aPTT-based actor IX activity remained stable from Year 1 (40.7%) up to Year 5 (46.7%) and mean ABR for the cumulative follow-up was 0.14 (Years 0-5) for all bleeds. A favourable safety profile was maintained over 5 years.²² Despite the limited population size, these data may provide further evidence of the long-term effects of HEMGENIX.^®21,22

About CSL
CSL (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, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise sav to e 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 29,000+ 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 CSL.com/Vita. For more information about CSL, visit CSL.com.

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Media Contacts Nordic Region

Helena Bragd, General Manager CSL Nordics

Mobile: + 46 (0)8 544 966 77

Email: Helena.GomerBragd@cslbehring.com

Fredrik Sjöö, MD PhD, Head of Medical Affairs CSL Nordics

Mobile: + 46 (0)8 544 966 85

Email: Fredrik.Sjoeoe@cslbehring.com

References

1. uniQure, Inc. CSL Behring GmbH. Hemgenix^®(etranacogene dezaparvovec): Summary of Product Characteristics [online]. Available at: https://www.ema.europa.eu/en/documents/product-information/hemgenix-epar-product-information_en.pdf [Last accessed: December 2024].

2. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26 Suppl 6:1-158. doi: 10.1111/hae.14046.

3. CSL Behring. CSL Behring Signs First Commercial Agreement in Austria to Fund Haemophilia B Gene Therapy HEMGENIX^®. Available at: https://www.cslbehring.de/en-us/news/2024/pm-hemgenix-agreement-austria. [Last accessed December 2024].

4. Medicinrådet. The Medical Council recommends the gene therapy Hemgenix following a new effect-based price agreement. Available at: https://medicinraadet.dk/nyheder/2024/medicinradet-anbefaler-genterapien-hemgenix-efter-ny-effektbaseret-prisaftale. [Last accessed December 2024].

5. National Institute for Health and Care Excellence. Final draft guidance: Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B. Available at: https://www.nice.org.uk/guidance/gid-ta10699/documents/674. [Last accessed December 2024].

6. Scottish Medicines Consortium. Etranacogene dezaparvovec (Hemgenix). Available at: https://scottishmedicines.org.uk/medicines-advice/etranacogene-dezaparvovec-hemgenix-full-smc2649/. [Last accessed December 2024].

7. Ministerio De Sanidad. Puntos destacados de la reunión de la Comisión Interministerial de Precios de los Medicamentos 26 de septiembre de 2024. Available at: https://www.sanidad.gob.es/areas/farmacia/precios/comisionInteministerial/acuerdosNotasInformativas/docs/NOTAINFORMATIVACIPM_SEPTIEMBRE2024.pdf [Last Accessed: December 2024].

8. Pipe SW, Leebeek FWG, Recht M, et al. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023;388:706-718. doi: 10.1056/NEJMoa2211644.

9. Spronck EA, Liu YP, Lubelski J, et al. Enhanced Factor IX Activity following Administration of AAV5-R338L "Padua" Factor IX versus AAV5 WT Human Factor IX in NHPs. Mol Ther Methods Clin Dev. 2019;15:221-231. doi: 10.1016/j.omtm.2019.09.005.

10. Thornburg CD. Etranacogene dezaparvovec for hemophilia B gene therapy. Ther Adv Rare Dis. 2021;2:26330040211058896. doi: 10.1177/26330040211058896.

11. Pipe S, van der Valk P, Verhamme P, et al. Long-Term bleeding protection, sustained FIX activity, reduction of FIX consumption and safety of hemophilia B gene therapy: results from the HOPE-B Trial 3 years after administration of a single dose of etranacogene dezaparvovec in adult patients with severe or moderately severe hemophilia B. Blood. 2023;142:1055.

12. Pipe SW, van der Valk P, Verhamme P, et al. Etranacogene dezaparvovec shows sustained efficacy and safety in adult patients with severe or moderately severe haemophilia B 3 years after administration in the hope-B Trial [EAHAD 2024 Oral Abstract OR09]. Haemophilia. 2024; 30: 25.

13. von Drygalski A, Pipe SW, Giermasz A, et al. Stable and durable factor IX levels over 4 years after etranacogene dezaparvovec gene therapy administration in a Phase 2b trial in patients with haemophilia B [EAHAD 2024 Abstract PO038]. Haemophilia. 2024; 30: 25.

14. Miesbach W, Recht M, Key N, et al. Durability of Factor IX activity and bleeding rate in people with severe or moderately severe haemophilia B after long-term follow-up in the phase 1/2 Study of AMT-060, and phase 2b and phase 3 studies of etranacogene dezaparvovec (AMT-061). Hamostaseologie 2023; 43: S46-S47.

EUR-HGX-0231 Mar 2026