MARBURG, GERMANY– September 30, 2024 – Global biotechnology leader CSL Behring (ASX: CSL) today announced it has been granted a positive recommendation by the Spanish Interministerial Commission on the Pricing of Medicines (CIPM) resulting in national reimbursement for HEMGENIX® (etranacogene dezaparvovec) for eligible haemophilia B patients in Spain.1
HEMGENIX® is the first one-time gene therapy approved in Spain for adults with severe and moderately severe haemophilia B (congenital Factor IX deficiency) without a history of Factor IX (FIX) inhibitors.2
This significant milestone underscores the value of HEMGENIX® and means that patients in Spain will have access to this groundbreaking gene therapy through the local healthcare system. This represents a paradigm shift, and transformative approach to haemophilia B management by offering a durable alternative to frequent intravenous Factor IX infusions, which can significantly impact the quality of life and wellbeing of patients.3
“We are proud that the Spanish CIPM has recognized HEMGENIX®, as an innovative one-time treatment option, marking a pivotal moment for Spanish haemophilia B patients and acknowledging the remaining unmet needs in haemophilia B treatment,” stated Maria Jose Sanchez Losada, General Manager, CSL Behring Iberia. “Following this positive recommendation, our priority will now be to work with healthcare authorities to conclude the agreement and ensure HEMGENIX® can reach patients and healthcare professionals throughout the country.”
CSL Behring is working with relevant stakeholders to continue the expansion of access to HEMGENIX® across Europe, building on recent milestone access decisions in UK, Scotland, Denmark, Austria and France.4-8
“The agreement with the CIPM for HEMGENIX® is an important step forward for Spanish patients, the local healthcare system and regulatory authorities,” said Diego Sacristan, Head of International, CSL Behring. “We are fully committed to continuing our close collaboration with stakeholders and local authorities to bring etranacogene dezaparvovec to all eligible people living with haemophilia B, where the unmet need still remains.”
About Haemophilia B
Haemophilia B is a life-threatening rare disease. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. 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.
About HEMGENIX®
HEMGENIX® is a 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 deficient protein in haemophilia B. It uses AAV5, a non-infectious viral vector, derived from an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating Factor IX proteins that are 5x-8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of Factor IX.
About the Pivotal HOPE-B Trial
The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Fifty-four adult haemophilia B patients classified as having moderately severe to severe haemophilia B and requiring prophylactic Factor IX replacement therapy were enrolled in a prospective, six-month or longer observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR). After the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5.
A total of 54 patients received a single dose of etranacogene dezaparvovec in the pivotal trial, with 52 patients completing at least three years of follow-up. The primary endpoint in the pivotal HOPE-B study was ABR 52 weeks after achievement of stable Factor IX expression (months 7 to 18) compared with the six-month lead-in period. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady-state Factor IX transgene expression. Secondary endpoints included assessment of Factor IX activity.
No serious treatment-related adverse reactions were reported. One death resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with etranacogene dezaparvovec by independent molecular tumour characterization and vector integration analysis. No inhibitors to Factor IX were reported.
Long-term three-year data presented at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) 2024 continue to reinforce the potential long-lasting efficacy and safety of etranacogene dezaparvovec and the ongoing benefit of this treatment for people living with haemophilia B.
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a 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 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 32,000 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.
Media Contacts
Stephanie Fuchs
Mobile: +49 151 584 388 60
Email: Stephanie.Fuchs@cslbehring.com
References:
1. 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: September 2024].
2. European Medicines Agency. First gene therapy to treat haemophilia B. Available at: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b. [Last accessed September 2024].
3. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26 Suppl 6: 1-158.
4. CSL Behring. NICE recommends CSL Behring’s HEMGENIX®. Available at:https://www.cslbehring.de/en-us/news/2024/pm-hemgenix-managed-access-uk. [Last accessed September 2024].
5. CSL Behring. SMC accepts CSL Behring’s HEMGENIX (etranacogene dezaparvovec) for use on an interim basis in Scotland. Available at: https://www.cslbehring.de/news/2024/pm-hemgenix-scottland. [Last accessed September 2024].
6. 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 September 2024].
7. 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 September 2024].
8. CSL Behring’s HEMGENIX® Granted First-Ever Direct Access in France for Haemophilia B Gene Therapy. Available at: https://www.cslbehring.de/en-us/news/2023/pm-hemgenix-direct-access-f. [Last accessed September 2024].
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