Latest news with #clotting
Daily Mail
18-06-2025
- Health
- Daily Mail
EXCLUSIVE 'I've dreamed of this moment': First ever patient gets 'world's most expensive drug' on the NHS - it costs £2.6million per dose
A 44-year-old man has become the first ever patient to receive a life-changing dose of the 'world's most expensive drug' on the NHS. Called Hemgenix, the drug—which costs an estimated £2.6million per patient—was administered yesterday at St Thomas' NHS Foundation Trust in London. Delivered as a one-off IV drip, it is the only treatment of its kind for haemophilia B. This is a bleeding disorder where the body doesn't make enough—or any—of a vital protein critical to clotting. Clotting stops wounds from bleeding, so those with the disorder risk suffering severe and even life-threatening blood loss from even minor injuries. Patients also run the risk of what are called 'spontaneous bleeds', which can be triggered without a direct injury and even prove deadly if they occur in a vital organ. Prior to Hemgenix, all haemophilia B patients needed regular weekly injections of an artificial clotting agent to keep their risk of catastrophic injury to a minimum. The patient, speaking anonymously, was diagnosed with haemophilia B when he was just 18 months old, and said he was now looking forward to a life 'free of worry'. 'I've always had to be more cautious and to plan ahead. There is a level of anxiety in that and being overly cautious has often led to missed opportunities and things I can't do, like contact sports,' he told MailOnline. 'To experience a life free of the worry and to do things that I wouldn't normally do will also be amazing.' He added: 'Not needing to plan ahead for treatment deliveries or looking up hospitals in foreign destinations when going on holiday, or having to tell people "sorry I can't do that I've got haemophilia", will be something I've always dreamed of.' Medics and charities have hailed the roll-out of the therapy as a critical milestone for helping those with the condition lead more fulfilling lives. Dr Pu-Lin Luo, the consultant haematologist at Guy's and St Thomas' who administered the treatment yesterday said it represented an 'exciting' step in treating the condition. 'This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients. 'It is also a testament to the advancement of cell and gene therapies in the UK,' she said. Kate Burt, chief executive of charity The Haemophilia Society, also added: 'Today marks an important milestone not just for this patient, but for all those living with haemophilia B in the UK.' 'The current treatment of lifelong intravenous injections can place a significant burden on those living with haemophilia and it has an impact on broader family, relationships and work. 'The availability of gene therapy for haemophilia B as a one-time infusion will allow those eligible for treatment to expand their horizons and live life to the full, free from the restrictions of regular injections.' Despite being approved for use on the NHS last year, yesterday's treatment is the first time Hemgenix has been used by the health service. It's also the first time the drug has been used in the UK outside of a clinical trial. Hemgenix is the brand name of the drug etranacogene dezaparvovec. It works by replacing a patient's defective gene—which is incapable of producing the clotting protein—with one that can, eliminating the need for regular injections. Studies on the gene therapy have shown the protective effect lasts for at least three years, but the hope is it could work for even longer. There are approximately 2,000 people with haemophilia B in the UK. Yet only around 260 with 'moderately severe or severe haemophilia B' are currently eligible for Hemgenix on the NHS. This puts the potential total bill to the taxpayer at about £676million. MailOnline understands, however, that the NHS acquired the drug at an undisclosed discount, so the actual cost is likely to be less. Additionally, while the cost of the drug is roughly £2.6million per patient, medics claim the treatment actually saves the NHS money in the long term. The lifetime cost of providing a patient the alternative weekly clotting injections has been estimated to be £8million. This sum doesn't include the cost of life-saving interventions and surgeries haemophilia B may also need. Hemgenix is made by Philadelphia-based pharmaceutical company CSL Behring. Reacting to the news today the firm's general manager for UK and Ireland, Eduardo Cabas, said: 'This patient receiving etranacogene dezaparvovec is a testament to the collaborative efforts of the haemophilia community, NICE and NHS England to ensure that patients in the UK are able to access this one-time treatment option.' Hemgenix was initially described as the 'most expensive drug in the world' when it originally came onto the scene in 2022. However, other drugs—also gene therapies—have since eclipsed it in terms of overall cost. Like all medications Hemgenix does come with the risk of side effects. Patients administered the drug take regular liver function tests to ensure the vital organ is functioning correctly. This is because there is a risk the medication could trigger a potentially dangerous immune response. These tests are held one a week for the first three months before they are reduced to once a year. Patient information leaflets distributed with the drug also highlight a potential increased risk of cancer due to the fact it inserts itself into the DNA of cells. However, clinical studies have—so far—shown no cancers have been caused by Hemgenix use. Those taking Hemgenix must also use contraceptives like condoms for at least a year following treatment, due to a process called 'shedding'—where the drug can be passed through bodily fluids like semen. Patients are also forbidden from donating blood for this reason.
Medscape
23-05-2025
- Health
- Medscape
NICE Recommends First Subcutaneous Option for Haemophilia B
The National Institute for Health and Care Excellence (NICE) has recommended marstacimab (Hympavzi, Pfizer) for treating severe haemophilia B. The treatment is advised for patients aged 12 years and over who weigh at least 35 kg and do not have factor IX inhibitors. It is the first subcutaneous injection for this condition. The treatment was approved by the Medicines and Healthcare products Regulatory Agency last month. How Marstacimab Works Marstacimab is administered weekly. It works by targeting tissue factor pathway inhibitor (TFPI), a protein that prevents blood from clotting. By reducing TFPI levels, marstacimab promotes the formation of thrombin, helping to increase clotting and stop bleeding. About 255 people in England live with severe haemophilia B, defined by a factor IX activity level below 1%. NICE estimates that around 205 patients will be eligible for marstacimab. People with haemophilia B currently receive factor IX replacement therapy by infusion, sometimes every 2-3 days. Gene therapy is also available, following approval by NICE last year. The final draft guidance is based on results from the BASIS study, a phase 3 trial that evaluated marstacimab in 116 adults and adolescents aged 12 years and older with severe haemophilia A or B without inhibitors. Marstacimab significantly reduced the annualised bleeding rate for treated bleeds during the 12-month active treatment period. It demonstrated both noninferiority and statistical superiority compared with routine factor-based prophylaxis. Not Recommended for Haemophilia A While marstacimab can also treat haemophilia A, NICE found it was not cost-effective for this group. It is therefore not recommended for NHS use in these patients. Current treatment options for haemophilia A include factor VIII, emicizumab, and efanesoctocog alfa. 'Marstacimab's clinical and cost-effectiveness compared with current treatment, together with its once-weekly dosing by injection under the skin, means it has the potential to significantly improve the quality of life of people with severe haemophilia B,' said Helen Knight, director of medicines evaluation at NICE. Conan McIlwrath, chair of the Haemophilia Society, welcomed the move. 'This will hopefully help people move towards more individualised treatment plans, based on what best supports the life they choose to live,' he said. Once the final NICE guideline is published, marstacimab could be available to eligible patients as early as this autumn.
Zawya
08-05-2025
- Health
- Zawya
New era for hemophilia acre in the UAE: Pfizer introduces first prefilled injectable therapy
Delivered through a once weekly prefilled autoinjector pen, offering greater convenience and independence for patients in the UAE DUBAI, UNITED ARAB EMIRATES — Pfizer announced today the launch of a new and innovative therapy for people living with hemophilia in the UAE. This marks a significant advancement in care for individuals affected by this rare and serious bleeding disorder. Hemophilia is a rare genetic bleeding disorder in which the blood fails to clot properly, leading to prolonged bleeding that can be either spontaneous or occur after an injury. Unlike traditional clotting factor replacements, Pfizer's new therapy targets the tissue factor pathway inhibitor (TFPI), a natural anticoagulant protein. By inhibiting TFPI, the treatment helps restore balance to the body's clotting process, reducing the frequency of bleeding episodes. This advanced therapy is administered once a week through a simple injection under the skin using a prefilled autoinjector pen. It is the first prefilled injectable therapy available in the UAE, designed to make treatment more convenient and accessible for patients, while reducing the burden of frequent infusions. Dr. Nadine Tarcha, Pfizer Gulf Medical Director, said: 'The introduction of this therapy reflects Pfizer's ongoing commitment to improving the lives of people with rare diseases. By combining scientific innovation with a more convenient delivery method, we aim to empower individuals with hemophilia to live with greater confidence and improved quality of life.' According to the World Federation of Hemophilia's 2023 Annual Global Survey, more than 32,000 in the Eastern Mediterranean region have been diagnosed with hemophilia. This represents less than half of the expected number of cases, suggesting that many individuals remain undiagnosed or lack access to appropriate care. These findings emphasize the importance of early diagnosis and patient-friendly treatment options that can support better long-term outcomes. The introduction of this therapy supports the UAE's broader healthcare vision, which prioritizes access to innovative treatments and improved quality of care for people living with complex and rare conditions. Patients are encouraged to speak with their healthcare provider to determine whether this treatment may be appropriate for their condition. About Pfizer: Breakthroughs That Change Patients' Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development, and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments, and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments, and local communities to support and expand access to reliable, affordable health care around the world.
