CSL Behring Announces First Two Patients Treated with HEMGENIX® (etranacogene dezaparvovec) Gene Therapy for Hemophilia B in Europe

MARBURG, Germany, July 04, 2024 (GLOBE NEWSWIRE) — Global biotechnology leader CSL Behring (ASX: CSL) today announced that two hemophilia B patients were treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec) at Hemophilia Treatment Centers in France. This milestone achievement makes HEMGENIX® the first gene therapy administered as a treatment in a real-world setting for hemophilia B in Europe.

HEMGENIX® is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe hemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors.1

Following European Commission approval, HEMGENIX® was the first ever therapy to be granted Direct Access in France2, thus enabling the first patients to be treated in Europe outside of the clinical program.

Though effective, current therapies can be time intensive and require regular treatment that can have a substantial impact on a patient’s daily life.3 HEMGENIX® offers a one-time treatment, allowing people living with hemophilia B to produce their own Factor IX, which can lower the risk of bleeding.4

“Only a few decades ago, gene therapy for hemophilia was a distant concept, which has now become reality. Accordingly, the first two patients treated with HEMGENIX® since receiving European approval is a major accomplishment and a testament to the joint commitment of the hemophilia B community, as well as the access and reimbursement authorities, in bringing innovative therapies to patients,” said Dr Lutz Bonacker SVP and General Manager, CSL Behring Commercial Operations Europe. “This milestone has been made possible by the innovative Direct Access scheme adopted in France, allowing patients to benefit from early access to pioneering treatments. We are encouraged to see increasing access to gene therapies in European countries and are fully committed to ensuring that access to potentially life-changing treatment continues.”

HEMGENIX® was granted conditional marketing authorisation by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the U.S. Food and Drug Administration (FDA) in November 2022. It has also been approved by Health Canada, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA), Switzerland’s Swissmedic and Australia’s Therapeutic Goods Administration (TGA).

The multi-year clinical development of HEMGENIX® was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment.

About Hemophilia B

Hemophilia 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 hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor.


HEMGENIX® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called 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 HEMGENIX®. Fifty-four adult hemophilia B patients classified as having moderately severe to severe hemophilia 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 HEMGENIX® at the 2×10^13 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 HEMGENIX® 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 HEMGENIX® 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 HEMGENIX® and the ongoing benefit of this treatment for people living with hemophilia B.

About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat hemophilia 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


1 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. [Accessed May 2024].
2 Republique Française. Légifrance: Article 62 of Law No. 2021-1754. Available at: https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000048551003 [Accessed May 2024].
3 Leebeek, F & Miesbach, W. (2021) Gene therapy for haemophilia: a review on clinical benefit, limitations, and remaining issues. Blood. Vol 138, Issue 11. pp923-931.
4 Coppens M et al. Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial. The Lancet Haematology 2024; 11(4):E265-E275.

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Minister of Defense Meets with Trkiye’s Defense Industrial Leaders, Witnesses Signing of MOUs

Istanbul: Minister of Defense Prince Khalid bin Salman bin Abdulaziz, as part of his official visit to the Republic of Trkiye, met on Wednesday with Defence Industry Agency President Haluk Gorgun and executives of large Turkish industrial companies.

Participants reviewed cooperation opportunities in the defense field according to Saudi Vision 2030.

Prince Khalid and Gorgun witnessed the signing of memoranda of understanding (MoUs) between Saudi and Turkish companies.

The signing ceremony was attended by high-ranking officials from the two countries.

Source: Saudi Press Agency

Lebanese Parliament Speaker, UN Official Discuss Situation in South

The Lebanese Parliament’s Speaker Nabih Berri, met Thursday with the United Nations Special Coordinator in Lebanon Jeanine Plasschaert.

The meeting discussed the situation in Lebanon and the region, in light of the Israeli entitys continued aggression against Lebanon and the Gaza Strip.

Source: Qatar News Agency

HH Shaikh Khalid bin Hamad emphasises Bahrain’s belief in youth

His Highness Shaikh Khalid bin Hamad Al Khalifa, His Highness Shaikh Khalid bin Hamad Al Khalifa, Supreme Council for Youth and Sports First Deputy Chairman, General Sports Authority (GSA) and Bahrain Olympic Committee President (BOC), affirmed that Bahrain is committed to supporting its youth and providing them with the conducive environment to achieve their aspirations. He highlighted the directives of His Majesty King Hamad bin Isa Al Khalifa and the support of His Royal Highness Prince Salman bin Hamad Al Khalifa, the Crown Prince and Prime Minister, as well as the efforts of His Highness Shaikh Nasser bin Hamad Al Khalifa, Representative of His Majesty the King for Humanitarian Work and Youth Affairs and Chairman of the Supreme Council for Youth and Sports (SCYS), all aimed at empowering youth to succeed and excel in various domains.

On the occasion of Arab Youth Day, celebrated annually on 5 July, His Highness extended congratulations to all Arab youth, emphasising that this day is an important opportu
nity to recognise the role of youth in building a brighter future for Arab societies and nation.

His Highness highlighted that Bahraini and Arab youth have always shown ambition and creativity, driving progress and development in all fields.

He stressed the importance of mobilising efforts from all governmental and private institutions to enhance initiatives aimed at empowering youth and providing opportunities to develop their skills and talents. His Highness urged the youth to take advantage of these opportunities and work diligently to achieve their goals and aspirations.

Source: Bahrain News Agency

Police Academy Celebrates Graduation of Two Groups of Tomorrows Police Program

The Police Academy on Thursday celebrated the graduation of the first and second groups of the tomorrows police program in its 5th edition (summer 2024), in the presence of HE Undersecretary of the Ministry of Interior Abdullah bin Khalaf bin Hattab Al Kaabi , and a number of Academy’s senior officers.

The graduation ceremony, which was held in the Academy’s sports hall, witnessed performances by graduates that highlighted the skills they acquired during the two-week program, including self-defense and air shooting skills, as well as various sports skills appropriate to age groups.

The ceremony also featured screening of a film about the tomorrows police program in its 5th edition, and another about the importance of awareness of the dangers of cybercrime and cybersecurity and how the competent authorities deal with them, in addition to a direct live show explaining the role of security personnel in combating drugs and following up and monitoring narcotic smugglers by sea.

In its 5th edition, the program t
argeted students born in (2012-2013-2014-2015) where they were divided into six groups. More than (900) students graduated in the first and second groups, after the training period that began on June 22nd, where as the rest of the groups will be graduated over the next few weeks.

The police of tomorrow program includes military and sports training. Military activities include infantry and air shooting, while sports activities include competitions, swimming, and self-defense, in addition to cultural and awareness activities and field visits in collaboration with a number of entities in the country.

It is noteworthy that this specific program aims to deepen national identity and the spirit of loyalty and belonging in the souls of young people, promote noble values and assume responsibility, contribute to community service and consolidate the authentic Islamic and Arab values, develop leadership skills, and enhance self-confidence, in addition to developing their skills and physical strength through military a
ctivities and sports.

Source: Qatar News Agency

HH Shaikh Nasser bin Hamad emphasises role of Bahraini youth

His Highness Shaikh Nasser bin Hamad Al Khalifa, Representative of His Majesty the King for Humanitarian Work and Youth Affairs and Chairman of the Supreme Council for Youth and Sports (SCYS), emphasised Bahrain’s belief in the vital role of its youth in advancing progress and achieving future development goals under the leadership of His Majesty King Hamad bin Isa Al Khalifa.

His Highness highlighted the significant attention given by His Majesty and other Arab leaders to empowering Arab youth, fostering genuine understanding and awareness, and enhancing their roles across various sectors to prepare them as future leaders.

In a statement on the occasion of Arab Youth Day, celebrated annually on 5 July, His Highness commended the Bahraini government’s efforts, led by His Royal Highness Prince Salman bin Hamad Al Khalifa, the Crown Prince and Prime Minister, in prioritising youth in the government’s agenda.

He noted the launch and execution of national programmes aimed at equipping the youth with the necess
ary skills and expertise to actively contribute to sustainable development across all domains.

His Highness elaborated that Bahrain has a clear, long-term vision for the future, relying on a unique methodology that involves youth in its realisation. This approach encourages the youth to envision and shape the future with their ideas and projects.

HH emphasised the commitment to instilling hope in Bahraini youth through opportunities for active engagement and communication, which motivates them to contribute effectively to national development.

His Highness Shaikh Nasser bin Hamad Al Khalifa expressed pride in the dedicated Bahraini youth who strive to elevate the nation’s status and achieve significant accomplishments, urging them to seize available opportunities to further develop their skills and abilities.

He called on them to serve as a model for Arab youth.

His Highness also commended the outstanding efforts of fellow Arab nations and the opportunities provided by the Arab League to Bahraini and Ara
b youth, which develops cooperation mechanisms within a collective Arab framework that aligns with the aspirations of empowering Arab youth.

Source: Bahrain News Agency