Once-weekly Altuviiio, a new kind of factor VIII therapy for haemophilia A that provides considerable bleed prevention, has been approved by the US FDA.

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Altuviiio [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], formerly known as efanesoctocog alfa, is a first-in-class, highly sustained factor VIII replacement therapy that has received FDA approval. For both adults and children with haemophilia A, the use of altuviiio is recommended for routine prophylaxis, on-demand treatment to stop bleeding episodes, and perioperative care (surgery). In comparison to previous factor VIII prophylaxis, bleeding is greatly reduced with altuviiio, the first and only haemophilia A medication that offers normal to near-normal factor activity levels (above 40%) for the majority of the week with once-weekly dose.

“Today’s approval of Altuviiio allows patients and physicians to rethink living with haemophilia,” stated Paul Hudson, CEO of Sanofi. Altuviiio’s ability to produce high sustained factor activity levels has the potential to alter the haemophilia landscape. For the first time, patients can have effective bleed prevention with a once-weekly dose. We have pledged to provide at Sanofi significant shifts in treatment paradigms that enhance people’s lives, like Altuviiio.

In the rare, lifelong disorder known as haemophilia A, the blood’s ability to clot properly is compromised, causing excessive bleeding and spontaneous bleeding into joints that may cause joint damage, persistent discomfort, and have an adverse effect on quality of life. The amount of clotting factor activity in a person’s blood determines how severe their haemophilia is, and the risk of bleeding is inversely correlated with factor activity levels.

“This approval marks an important clinical advancement for the haemophilia community because we have an option that can achieve higher levels of factor activity with a single, simplified weekly dose,” said Lynn Malec, MD, medical director of Comprehensive Center for Bleeding Disorders, Associate Investigator at The Versiti Blood Research Institute, and Associate Professor of Medicine and Paediatrics at The Medical College of Wisconsin. Patients may trust the bleed protection offered by Altuviiio since it maintains high levels of factor activity throughout the week.

This is Altuviiio’s first endorsement. The FDA reviewed the application under Priority Review, a designation given to medical treatments with the potential to significantly advance the diagnosis, treatment, or prevention of critical illnesses. The FDA previously recognised Altuviiio as a Fast Track drug in February 2021, an Orphan Drug in August 2017, and a Breakthrough Treatment in May 2022 (making it the first factor VIII medication to receive this classification).

In the second half of 2023, a regulatory filing in the EU is anticipated. In June 2019, the European Commission authorised the designation of orphan drugs. By extending protection, Altuviiio helps raise expectations for haemophilia A. The key XTEND-1 phase 3 research results, which were just published in The New England Journal of Medicine, served as the basis for the FDA’s clearance. Once-weekly Altuviiio prophylaxis achieved the primary goal, significantly reducing annualised bleeding rates (ABRs) for individuals with severe haemophilia A, with a mean ABR of 0.70 (95% CI: 0.5-1.0) and a median ABR of 0.0. (Q1, Q3: 0.0, 1.0). Based on an intra-patient comparison (95% CI:58%-87%), Altuviiio met the primary secondary goal with a substantial reduction of 77% in ABR compared to previous factor prophylaxis.

Further information demonstrated that joint bleeding was prevented, with a median yearly joint bleeding rate of 0. (Q1, Q3: 0.0, 1.0). Target joints, or joints with recurring bleeding, completely disappeared after treatment with Altuviiio (e.g., knee, ankle, or elbow). With a mean factor VIII activity of more than 40% for the most of the week and more than 10% on Day 7, Altuviiio offered a low bleed risk. In the study, Altuviiio was well tolerated, and although it is possible after administration of Altuviiio, factor VIII inhibitor development was not found.

Furthermore, preliminary data from XTEND-Kids revealed that children under the age of 12 who received once-weekly Altuviiio for 26 weeks (n=23) had a mean ABR of 0.5 (95% CI: 0.2-1.3) and a median ABR of 0. (Q1, Q3: 0.0, 1.3). Data from the XTEND-1 trial were consistent with the safety findings. A future medical symposium will feature a presentation of the complete XTEND-Kids results. Altuviiio has a well-established safety profile across trials, and while the development of factor VIII inhibitors has not been reported, it is feasible after the administration of Altuviiio. Headache and arthralgia are the side effects of Altuviiio that occur the most frequently (>10%).

Regular prophylaxis, on-demand therapy and management of bleeding episodes, and perioperative management of bleeding are all advised for the use of altuviiio. All patients and a variety of clinical situations are intended to be treated with the same prescribed dose of 50 IU/kg. Sanofi will price Altuviiio at parity to the annual expenses associated with treating a prophylaxis patient on Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] in order to guarantee that patients have access to the increased bleed prevention offered by Altuviiio. Moreover, Sanofi will offer extensive patient support services and tools online and at 1.855.My Altuviiio (855.692.5888). Altuviiio is anticipated to go on sale in the US in April.

For both adults and children with haemophilia A, Altuviiio [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] is a novel von Willebrand factor (VWF) independent recombinant factor VIII therapy that aims to prolong protection from bleeding with once-weekly preventive dose. In comparison to factor VIII products with conventional and extended half-lives, Altuviiio has a 3–4 fold longer half-life. The von Willebrand factor ceiling, which places a half-life restriction on earlier generation factor VIII therapies, has been demonstrated to be overcome by this factor VIII therapy for the first time. By including a portion of von Willebrand factor and XTEN polypeptides to lengthen its duration in circulation, Altuviiio expands on the ground-breaking Fc fusion technology.

The XTEND clinical programme consists of two haemophilia A Phase 3 trials: XTEND-1 in adults over the age of 12 and XTEND-Kids in children under the age of 12. A follow-up study is also being conducted (XTEND-ed). In the phase 3 XTEND-1 research (NCT04161495), participants 12 years of age and older with severe haemophilia A who had previously received factor VIII replacement therapy were evaluated for safety, effectiveness, and pharmacokinetics of once-weekly Altuviiio. The study included two parallel treatment arms: the prophylaxis Arm A (n=133), which treated patients who had previously received factor VIII prophylaxis with once-weekly intravenous Altuviiio prophylaxis (50 IU/kg) for 52 weeks, and the on-demand Arm B (n=26), which treated patients who had previously received on-demand factor VIII therapy with 26 weeks of on-demand Altuviiio (50 IU/kg) before switching to once.

The key secondary endpoint was an intra-patient comparison of ABR during the Altuviiio weekly prophylaxis treatment period versus the prior factor VIII prophylaxis ABR for a subset of participants in Arm A who had participated in a prior observational study (Study 242HA201/OBS16221). The primary efficacy endpoint was the mean annualised bleeding rate (ABR) in Arm A. An open-label, non-randomized interventional research called XTEND-Kids (NCT04759131) examined the safety, effectiveness, and pharmacokinetics of once-weekly Altuviiio in individuals with severe haemophilia A who had previously received treatment (n=67). For 52 weeks, patients got 50 IU/kg of Altuviiio prophylaxis once weekly.

Alprolix and Elocta/Eloctate are being developed and marketed under a partnership between Sobi and Sanofi. The businesses also work together to develop and market efanesoctocog alfa, commonly known as Altuviiio, in the US. In the Sobi area, Sobi has exclusive development and commercialization rights (essentially Europe, North Africa, Russia and most Middle Eastern markets). With the exception of the Sobi territory, Sanofi owns the final development and commercialization rights in North America and everywhere else in the world. The lives of people with uncommon diseases are being transformed by Sobi, a specialist multinational biopharmaceutical company.

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