Sanofi Announces Phase III XTEND-Kids Data for Once-Weekly ALTUVIIIO in Children with Severe Hemophilia A
Sobi
Rare Disease Drug Provider, Specialty Drug Developer and Marketer
Sanofi
Pharmaceutical R&D Developer
On July 17, according to the Sanofi website, its jointly developed ALTUVIIIO with Sobi (Efanesoctocog alfa) The results of the Phase III XTEND-Kids study conducted in pediatric patients were published in The New England Journal of Medicine (NEJM), demonstrating the efficacy, safety, and pharmacokinetic properties of ALTUVIIIO.
Screenshot source: Sanofi official website
ALTUVIIIO is a first-of-its-kind, highly persistent Factor VIII replacement therapy. The drug, through its unique Fc-VWF-XTEN fusion protein design, extends the circulation time in the body, allowing for once-weekly dosing.
The XTEND-Kids study enrolled 74 previously treated patients under the age of 12 with severe Hemophilia A. Patients received ALTUVIIIO once weekly.50 IU per kilogram of body weight) Preventive treatment, lasting 52 weeks.
The study results showed that no factor VIII inhibitors were detected during the treatment process (0%，95%CI：0-5%). Among the 73 patients treated according to the protocol, the annualized bleeding rate (ABR) The median is 0.00 (IQR：0.00-1.02), with an estimated average ABR of 0.61 (95%CI：0.42-0.90）。
The study further showed that 64% of children had no untreated bleeding episodes, and 88% had no spontaneous bleeding. Preventing joint bleeding is crucial for maintaining joint health. In the study, 82% of children did not experience joint bleeding, indicating that ALTUVIIIO is expected to provide long-term protection for joint health.
In terms of safety, ALTUVIIIO demonstrated good tolerability in children, with no treatment discontinuations due to adverse events, and no serious allergic reactions or embolic or thrombotic events were reported.
Moreover, in the pediatric population, due to the high clearance rate of administered factor in the blood, it is usually necessary to inject standard half-life (SHL) or extended half-life (EHL) Factor VIII products 2-4 times per week. In contrast, the once-weekly dosing regimen of ALTUVIIIO significantly reduces the treatment burden on patients, demonstrating its remarkable advantages in clinical application.
ALTUVIIIO is the first Factor VIII therapy to receive FDA Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation. The European Commission (EC) also granted it orphan drug designation in June 2019.
Currently, ALTUVIIIO has been approved for marketing in the United States and Japan. On June 17 this year, the EC approved the drug for routine prophylactic treatment and on-demand treatment to control bleeding episodes in adult and pediatric patients with Hemophilia A, as well as for perioperative management.
In China, ALTUVIIIO entered Phase III clinical trials in November 2020 and is expected to complete the trial by January 2027. We look forward to future data releases providing further academic support for the efficacy and safety of this treatment.
Screenshot source: Insight Database official website
Regarding the conclusions of this study, Dr. Dietmar Berger, Global Head of Development and Chief Medical Officer at Sanofi, stated that the XTEND-Kids study data further confirms the significant association between high sustained factor activity levels and improved health outcomes, particularly joint health. Providing effective bleed protection treatment options for children with hemophilia not only enhances their quality of life but also offers greater peace of mind and assurance for families in their daily activities.
Reference Content:SanofiOfficial website (https://www.sanofi.com/en/media-room/press-releases/2024/2024-07-17-21-27-40-2914957）
