Latest news with #clotting
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 The final draft guidance is based on results from the BASIS study, a phase 3 trial that evaluated marstacimab in 116 adults and adolescents aged 12 years and older with severe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.
