FDA accepts sBLA for ALTUVIIIO in children with hemophilia A

FDA accepts sBLA for ALTUVIIIO in children with hemophilia A | Image Credit: © Postmodern Studio - © Postmodern Studio - stock.adobe.com.

The FDA has accepted a supplemental biologics license application (sBLA) for ALTUVIIIO (Antihemophilic factor; Sanofi) for managing bleeding in patients aged 12 years and younger with severe hemophilia A.

Hemophilia A impairs a person’s blood from being able to clot properly, causing excessive and spontaneous bleeding. This can lead to join damage and chronic pain which adversely impact quality of life. Patients are often diagnosed with hemophilia at a young age, with severity measured based on the level of clotting factor activity.

ALTUVIIIO is a high-sustained factor 8 therapy taken once a week by patients with hemophilia A for routine prophylaxis and on-demand treatment to control bleeding episodes as well as surgery. Compared to standard factor 8 products, ALTUVIIIO’s half-life is increased 3 to 4 times. Children taking the product weekly achieve activity levels within or near the normal range for approximately 3 days.

Most factor 8 therapies have a limitation on their half-life from the von Willebrand factor ceiling, but ALTUVIIIO has been shown to break through this barrier. In the phase 3 XTEND-Kids trial (NCT04759131), the safety, efficacy, and pharmacokinetics of ALTUVIIIO in previously treated patients aged under 12 years of age with severe hemophilia A were evaluated.

ALTUVIIIO 50 IU/kg was given to patients once per week for 52 weeks. The median and mean annualized bleeding rates (ABRs) in participants receiving weekly treatment with ALTUVIIIO were the primary endpoints of the analysis. Both endpoints were met during the trial, with median and mean ABRs of 0 and 0.89.

Safety and efficacy were confirmed, with no severe allergic reactions, anaphylaxis, or embolic or thrombotic events occurring in the study population following treatment. Common treatment-related adverse events included positive COVID-19 test, upper respiratory tract infection, and fever.

Following these results, the FDA accepted the sBLA for ALTUVIIIO. The Prescription Drug User Fee Act date is May 10, 2024.

Reference

ALTUVIIIO supplemental biologics license application based on positive final results from Phase 3 XTEND-Kids study accepted by FDA. Sanofi. September 12, 2023. Accessed September 12, 2023. https://www.news.sanofi.us/2023-09-12-ALTUVIIIO-R-supplemental-biologics-license-application-based-on-positive-final-results-from-Phase-3-XTEND-Kids-study-accepted-by-FDA