Introduction
Landmark Approval of Haemophilia A Therapy Hailed by Professor Klamroth The European Commission has granted marketing authorisation for a new, high-efficiency treatment for Haemophilia A, a development that is being called a major turning point by the medical community, led by key researcher Professor Robert Klamroth. The newly approved therapy, efanesoctocog alfa (marketed as Altuvoct), represents a significant advance in prophylactic care for patients with Haemophilia A, an inherited bleeding disorder caused by a deficiency in Factor VIII clotting protein. The decision, following a positive opinion from the European Medicines Agency (EMA), offers patients a once-weekly intravenous treatment option that clinical trials demonstrate provides dramatically superior Factor VIII activity protection compared to conventional therapies. Professor Klamroth, a distinguished haematologist involved in the pivotal studies, stated that the approval heralds a more ambitious and quality-of-life focused era for managing the condition. Overcoming Clinical Limitations Haemophilia A patients traditionally rely on prophylactic infusions of Factor VIII, which has a short half-life and requires frequent injections, often two or three times a week, to prevent spontaneous bleeding episodes. A key limitation of previous treatments has been the natural regulation of Factor VIII by the von Willebrand factor (VWF) protein in the bloodstream, which restricts how long the therapeutic factor remains active at high levels. Efanesoctocog alfa is designed to decouple the therapeutic Factor VIII from the VWF clearance mechanism, fundamentally extending its half-life. By circumventing this natural limitation, the drug allows for the maintenance of high, near-normal Factor VIII activity levels (above 40%) for a significant portion of the week. This is a critical factor, as maintaining higher 'trough levels' is directly correlated with better protection against debilitating joint bleeds, the most common complication of severe haemophilia.
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Professor Klamroth, who is the Head of the Department for Internal Medicine and Haemostasis at Vivantes Klinikum in Berlin, emphasised the philosophical shift this represents in treatment objectives. "For decades, the goal was merely to prevent life-threatening bleeds," he told reporters. "But now, the treatment goal has shifted; we wanted to be more ambitious. Maintaining sustained Factor VIII activity above 40% opens the door to a genuinely non-haemophilic lifestyle. " Patient Burden and Quality of Life The practical implications for patients are profound. The primary clinical benefit highlighted by the research is the reduction in treatment frequency from multiple weekly injections to a single, once-weekly intravenous infusion. This dramatically lowers the treatment burden, especially for adolescents and working adults, and fosters greater treatment adherence. The approval was based on the successful results of two international, multi-centre clinical trials, XTEND-1 (for adults and adolescents) and XTEND-Kids (for children). The XTEND-1 trial, which enrolled 159 patients across 48 centres globally, demonstrated that once-weekly prophylaxis with efanesoctocog alfa resulted in significantly reduced annual bleeding rates (ABR).
The mean ABR for treated patients was clinically low, reinforcing the therapy's efficacy. “A once-weekly treatment offers patients a much lower treatment burden with better protection and is more convenient for them," Professor Klamroth noted, adding that feedback from both patients and clinicians involved in the trials was overwhelmingly positive. The reduction in injection frequency, coupled with superior bleed protection, is expected to result in a considerable increase in the overall quality of life, allowing individuals greater spontaneity in daily life, exercise, and travel. Klamroth’s Context and the Future Landscape Professor Klamroth’s involvement in the efanesoctocog alfa trials underscores his position at the forefront of European haematology research. As the former President of the European Association for Haemophilia and Allied Disorders (EAHAD), he has consistently championed the adoption of novel, high-impact therapies aimed at minimising long-term morbidity associated with bleeding disorders. His clinic's philosophy has long been one of proactive prophylaxis, starting in early childhood, with the goal of preventing joint atrophy and chronic disability. The approval arrives at a time of rapid innovation in haemophilia management. While efanesoctocog alfa provides a substantial improvement for intravenous factor replacement, the field is also exploring non-factor therapies, such as the bispecific antibody emicizumab, and potentially curative approaches like gene therapy. Analysts suggest that Altuvoct’s approval positions it as the new benchmark for high-efficiency, standard-of-care factor replacement.
The new therapy complements rather than replaces the gene therapy research, which is still navigating regulatory and long-term efficacy hurdles. Outlook for Haemophilia Care The granting of marketing authorisation for efanesoctocog alfa marks a definitive step towards achieving therapeutic parity with individuals without haemophilia. For Professor Klamroth, this development validates years of research focused on extending the therapeutic window of Factor VIII. "From my point of view, it is great progress," Professor Klamroth concluded. "For intravenous treatment, replacing the missing clotting factor is a great step forward because patients have higher levels, better protection, and the convenience of once-weekly injections. " As the therapy rolls out across European member states, the focus will shift to ensuring equitable access and integrating this advanced prophylactic option into existing national haemophilia care infrastructures, securing a brighter outlook for thousands of patients.
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