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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Ayorkor Botchwey promotes AfCFTA at Namibian Trade Event

Ghana’s Foreign Minister, Ms. Shirley A. Botchwey, has made an impassioned case for the African Continental Free Trade Area (AfCFTA), to an audience of hundreds of business leaders in Namibia, and two southern African Presidents and members of their cabinets.

Speaking as a special guest at the opening of the Sixth Swakopmund International Trade Expo (SWAiTEX) Ms. Botchwey said: ‘It is our responsibility as leaders in government, industry, finance, SMEs, startups, regulation and civil society to harness our collective resources and capabilities towards an efficient market in trade and services, job creation, and prosperity for all Africans.’

The event in Swakopmund, 360km west of the capital, Windhoek, was attended by Presidents Nangolo Mbumba of Namibia, and Mogkweetsi Masisi of Botswana whose delegation included his Vice President Slumber Tsogwane and, in his own words, ‘half of my cabinet because we want to trade; we want to do serious business.’

‘The African Continental Free Trade Area is the beacon of
hope for Africa’s economic resurgence, Ms. Botchwey said, quoting the South African AfCFTA Secretary-General, Wamkele Mene. ‘We agree wholeheartedly, she added.

Resplendent in an African-print attire and hand-woven kente shawl over her shoulders, Ms. Botchwey, a former deputy trade minister, said Africans must make ‘African products our preferred products,’ while building partnerships regionally and across the continent to service the common market.

As a free trade area, connecting 1.3 billion people across 55 countries, with a combined Gross Domestic Product (GDP) of US$3.4 trillion, she said the AfCFTA, which is headquartered in Ghana, promises immense new markets, accelerated industrialization and fresh investment opportunities.

The theme for the expo is ‘Efficient Connectivity and Resource Beneficiation for Sustainable Growth in Africa.’

Ms. Botchwey acknowledged that African economies were currently facing challenging headwinds, including cascading impacts on debt, energy, food security, cost of livi
ng, unemployment and climate crises.

However, she pointed out that ‘the transformative potential of the AfCFTA shows that we can overcome our challenges and build resilient economies.’

She said Ghana was pleased that creative initiatives were being introduced to expedite trading under the AfCFTA, including payments settlements.

She commended Namibia for recently joining an AfCFTA-Guided Trade Initiative with Ghana, South Africa, Kenya, Tanzania and Cameroon.

‘This affirms your commitment and political will for meaningful trade within the African continent,’ Ms. Botchwey told President Mbumba who assumed office in February following the death of President Hage Geingob

The Guided Trade Initiative was established in 2022 as an interim measure to begin trade among interested parties, once they have met the minimum threshold for trading under the AfCFTA.

It is designed to test the Agreement’s capacity to function as envisaged, and indentify and fix imperfections.

A Memorandum of Understanding was also signe
d in April 2023 between the Ghana Investment Promotion Centre and the Namibia Investment Promotion and Development Board to facilitate investment and trade flows between the two countries, under the umbrella of the AfCFTA.

Ms. Botchwey, a candidate for Commonwealth Secretary-General, has made trade and investment a major plank of her vision. Namibia, Botswana and Ghana are among Africa’s 21 Commonwealth countries. There are 56 of them altogether.

She said developing countries with overlapping memberships in multilateral institutions must work together consciously and strategically to change their marginalised status in global trade and governance.

Source: Ghana News Agency

COCOBOD CEO inspires cocoa farmers on best practices to increase yield

Mr Joseph Boahen Aidoo, the Chief Executive Officer (CEO) of Ghana Cocoa Board (COCOD), has inspired and educated cocoa farmers in the Central Region on best practices to increase yield.

This was at two separate durbars, attended by more than 500 farmers at Gomoa Afransi and Ajumako, after a visit to some farms in the Armah, Jacob, and Sampreko communities, defying the heavy rains on Wednesday, July 3.

Following his visit, observation and interaction with the farmers, Mr Aidoo was concerned that many of the farmers were producing below their capacities, citing a lack of adherence to best farming practices.

Addressing the farmers, he said while the farms could produce high yields of cocoa, many farmers were producing an average of three to four to four bags per hectare, instead of about 10 bags per hectare.

He said: ‘Ordinarily, farmers shouldn’t have less than 1,000 kilograms (10 bags) per hectare, but observed that farmers are doing 450kg to 600kg (4.5 to six bags), which is very low.’

‘During the visit
, we observed that some of you [farmers] were doing slash and burn; it’s outmoded and not a good practice. After weeding, don’t burn it; both the weeds and the branches of the pruned trees are food for the soil,’ he said.

‘There are some microorganisms in the soil that we don’t see with our eyes, but they provide a lot of nutrients to the cocoa tree. Once you burn on your farm and use weedicides like ‘run up’ and ‘condemn’ – glyphosate, you kill them,’ he noted.

He explained that those weedicides contained acid and other chemicals, which were harmful to the cocoa and other plants, killed microorganisms and made soils lose their essential nutrients, thereby, reducing crop yields.

Once the cocoa and other food crops absorb those chemicals, it was transferred to the fruits, which were then eaten by humans, causing liver and kidney cancers, and slowly killing people, Mr Aidoo explained.

He recommended the use of poultry manure, as well as the use of the weeds and pruned branches of cocoa trees as mulch.

He a
dvised them to constantly prune their cocoa trees to enable them to bear flowers, which is critical to the survival of the trees producing more fruits, and preventing the crops from being attacked by pests.

Taking turns to engage with the COCOBOD team, the farmers appealed to the government for good road networks, affordable spraying machines and approved fertilizers.

Responding to their request, Mr Aidoo said though COCOBOC could not promise the construction of health centres, it would in about two months reach out to farmers through its cocoa clinic to screen, and assist, particularly, extension officers.

He said the durbar was an opportunity to intensify public education and sensitisation on acceptable farming and modern practices, which would be beneficial to farmers and their farms, as well as the country.

It also helped in assessing the results of extension officers and government interventions and what more ought to be done to educate, and support farmers to help increase cocoa yields.

He explaine
d that because of the technicalities involved in the fieldwork of the extension officers to farmers, it was important to decode their education in local languages for the farmers to easily comprehend and apply.

The officers themselves learn a few communication techniques through the presentations and public fora to complement their predominantly English language technical work, Mr Aidoo said.

Source: Ghana News Agency

PARM sensitises private sector stakeholders on agriculture risk management

The Platform for Agricultural Risk Management (PARM), in collaboration with the Italian Agency for Development Cooperation (AICS), has organised a trainer’s workshop for stakeholders in the private sector. 

The participants included farmer organisations, exporters, processors, buyers, enterprise federations, women’s organisations, chambers of commerce, and microfinance institutions.

Representatives from PARM donors and their projects, as well as its partners, also participated in the three-day event, which aimed to build the capacity of participants in risk management through a holistic approach, risk assessment and prioritisation by value chain and evidence-based risk tools and strategies, and monitoring and evaluation.

The training programme also featured interactive exchanges and group work, which allowed PARM and its partners to share best practices for managing risks in key value chains and explore synergies in the agricultural sector.

Miss Francesca Nugnes, the Capacity Development Specialist at PAR
M, and a trainer underscored the relevance of the programme, adding that it would help the private sector to plan the allocation of their resources in the midst of crises.

‘Essentially, planning and managing agriculture risk gives the private sector the ability to plan and manage how they will allocate resources and derisk any investment done. The objective of the training is to have a better view of agriculture risk, be aware of the risk, and learn how to manage it. We have done this in collaboration with agencies from Italy, and we have engaged all the private sectors along the value chain on how they can address the risk in their sectors.

This training programme has a lot of impact due to the synergy it creates between the private sector in the agriculture value chain,’ she said.

She also noted that the training programme also allowed the private sector to create partnerships with other countries, especially Italy. 

Mrs. Georgina Koomson, the Chief Executive Officer for Provident Farms, said the traini
ng programme would help her company diversify its operations.

She said the training also afforded her the opportunity to leverage her experiences and share them with other colleagues from different organisations.

Source: Ghana News Agency

EU investors urged to foster lasting ties with local suppliers

Mr Kobina Tahiru Hammond, the Minister for Trade and Industry, has urged European Union investors in Ghana to foster lasting ties with local suppliers to develop a reliable and sustainable supply chain. 

The Minister urged the investors to emulate their counterparts in Ghana, who have created and sustained lasting ties with local suppliers, which had engendered a consistent supply of raw materials. 

‘There are already several companies that are doing a yeoman’s job of connecting with local suppliers. Three industrial giants in the food and beverage industry that come to mind are Guinness Ghana Limited, Kasapreko Company Limited, and Nestle Ghana Limited, who have shown the way by building quite effective supplier development programmes involving farmers and aggregators of millet, sorghum, and maize.

This has been beneficial for local companies such as Premium Foods and Yedent Agro Limited, which have developed robust sub-contracting supply linkages with some of these large manufacturing industries,’ he sai

The Minister said this when he opened the second Ghana-European Union Business Forum, which was held in Accra. 

The forum was under the theme: ‘Fostering an Investment in Non-Traditional Chains Under the EU Global Gateway Strategy.’

He said the EU was  one of the largest investors in Ghana, with investments spanning various sectors, notably agriculture, energy, manufacturing, infrastructure, telecommunications, financial services, and pharmaceuticals.

These investments not only support Ghana’s industrial transformation agenda but also contribute in many ways to economic growth and sustainable development, he said. 

The Minister said the EU was one of the most successful regional blocks, which holds many lessons for African Continental Free Trade ambitions.

Mr Irchad Razaaly, EU Ambassador to Ghana, said the EU Global Gateway Strategy, in force since December 2021, offered a unique opportunity for Ghana and the EU to strengthen their economic ties, foster innovation, and create sustainable value chai

He said that by exploring non-traditional sectors, the EU and Ghana could unlock new opportunities, address challenges, and promote economic transformation. 

The Ambassador said the EU aimed to pursue economic cooperation with Ghana to support more value-added products exported from Ghana to transcend the traditional raw materials to the EU and the world. 

He noted that when both countries join forces, they would expand their reach, enhance results, and promote shared learning to  create unique and innovative products. 

According to the Trade Ministry, in 2023, exports from Ghana saw a promising eight per cent increase, rising from pound 2.4 billion to pound 2.6 billion Euros, while imports from the EU to Ghana experienced an 11 per cent decline, dropping from 3.7 billion Euros to 3.3 billion Euros. 

The total trade between Ghana and the EU in 2023 amounted to six billion euros, marking a slight decrease of three per cent compared to 2022. 

The trade flows between Ghana and the EU are characterised
by Ghana exporting primarily raw and semi-processed goods while importing machinery, vehicles, and chemicals.

Source: Ghana News Agency

Ghanaian youth encouraged to leverage opportunities of AfCFTA

Mr Kojo Adu, Head, Partnerships and ResourceMobilisation Unit, Ghana Technical and Vocational Education and Training (TVET) Service, has encouraged Ghanaian youth to leverage the business opportunities offered by the African Continental Free Trade Area (AfCFTA).

He noted that with the over 1.4 billion population of Africa, young people had a huge market size that they could explore to build successful businesses and speed up the development of Ghana and the continent at large.

He emphasised the continental median age between 18 and 22 years, saying young entrepreneurs should explore that youthful age group and produce strategic business ideas to cater for the needs of such group.

Mr Adu pointed out that, whilst AfCFTA offered a wider market, it also exposed Ghana to competition from other African countries.

That, he said, required Ghanaian youth to build relevant skills and expertise to deliver top-notch products and services to remain and thrive in business.

Mr Adu said this at the 15th Students for the
Advancement of Global Entrepreneurship (SAGE) National High School Entrepreneurship Competition held at the University of Ghana Business School.

The event, which seeks to nurture the leadership and entrepreneurial abilities of young people, saw Aburi Girls’ Senior High School winning the 2024 edition.

Mr Adu advised the youth to build trust in business, especially by keeping their word anytime they promised to deliver a product or service at a specific time and quality.

Dr Peter Gash, a mining consultant, said Ghana must strengthen its apprenticeship system across all fields in the educational sector so that irrespective of one’s academic qualification, every graduate would undergo apprenticeship to fit properly into the world of work.

He urged the Ghana government to cut down the long bureaucratic processes that usually delayed and stifled new startups from flourishing.

Dr Gash asked young people to show commitment to business and to take their studies seriously, saying, ‘Education is freedom. Treasure
it, because it is your passport to the rest of the world.’

Mr Festus Owooson, Director of Migration Advocacy Centre, encouraged the youth to form partnerships, leverage the collaborative strengths of one another, and allow themselves to be mentored to grow their enterprises into world-class businesses.

Mrs Eunice Dewi Adjei, Project Coordinator, WERise Network, said the country must create an inclusive environment to foster the spirit of entrepreneurship in both males and females.

She urged young people to build resilience, acquire knowledge and skills, develop a success mindset, cultivate self-confidence, and utilise digital tools for positive ends.

Source: Ghana News Agency